A burst of new drug approvals at the FDA just before Christmas pushed the total to 41 for 2014, the fastest pace in the past 18 years and a fresh sign that at least a few of the lead players have learned how to efficiently target new therapies for development.
Added together, 2014 marked the second highest year on record for the approval of new chemical entities, topped only by the 53 recorded at the FDA in 1996. The tally far exceeded last year's anemic pace of 29 new drug approvals, which in turn followed a spike of 39 new OKs in 2012. Last year's approval pace was also well ahead of the annual average of 24 new drug approvals for the first 10 years of the millennium and easily tops the 31-per-year average marked in the 1990s.
But while the FDA has also proved particularly eager to sanction new "breakthrough" drugs--9 for last year compared to just 3 in 2013--there's a caveat to the year's encouraging performance. A number of these new drugs were hurried along to fill a gap being left by blockbusters which either lost or will soon lose their patent protection. And they're arriving on the market as high prices have spurred a furious counterattack among private payers in the U.S., raising serious questions about the new generation's long-term commercial prospects.
"It's the best year since 1996, which is encouraging," notes R&D consultant Bernard Munos in an end-of-the-year assessment for FierceBiotech. "Even more so are the cure rates and remission rates, and what is coming up in the pipeline (CAR T-cell, CRISPR, bioprinting, tissue engineering, etc). It suggests that this spike, unlike previous ones, is likely to be long-lived. Have we turned the corner? The firms that have had the foresight and the courage to stray from their comfort zones and embrace the translation of these breakthrough discoveries are certainly on a strong footing. The others will continue to struggle."
Not everyone got to share the wealth of NDAs this past year. Pfizer ($PFE), which carved several billion dollars out of its research budget in the past 4 years, was left out of the winners' circle. Its absence underscores a weak pipeline that can't be entirely camouflaged by the prospects of such promising therapies as palbociclib.
Some Big Pharma players managed to break a long drought in the clinic with some key approvals. Eli Lilly ($LLY) scored a few new OKs from the FDA, topping the lot with a green flag for dulaglutide. Merck ($MRK) got a pioneering OK for Keytruda but had a far less enthusiastic reception for suvorexant, its sleep drug that was approved at a far lower dosage than the company was looking for. And AstraZeneca scored 4 approvals, putting it on top and giving it boasting rights to a badly needed record of success after years of turmoil.
Gilead ($GILD) got its big break with Harvoni, the combo follow-up to Sovaldi, then AbbVie ($ABBV) came along with its own cocktail, Viekira Pak. Every big breakthrough in drug development--like new interferon-free cocktails for hepatitis C, or PD-1/PD-L1 in cancer or the GLP-1 diabetes drug class--attracts a host of competitors. Big Pharma has a habit of traveling in packs.
While Harvoni exemplifies the immediate market potential of a major new treatment, it also provides a cautionary tale for the industry. AbbVie may have a less convenient dosing regimen for its hep C drug, but payers' outrage over Gilead's handling of its steep price for Sovaldi and later Harvoni gave AbbVie an opening to trade a discount price in exchange for an exclusive place on the key formulary at Express Scripts. AbbVie's approval also marks the first major OK for the company in the past decade, with huge implications for the company as it faces the loss of patent protection on its flagship product, Humira.
Discount pricing is one trend that will only grow more pronounced in the coming years, as payers use all the leverage at their disposal to shave costs, and it will test some of those blockbuster peak sales estimates some analysts like to attach to new drugs. Eli Lilly, for example, delivered the group of NDAs it promised investors--after reneging on a vow to get started in 2013--but now has to prove that these new drugs can deliver major sales revenue. Approvals aren't enough to determine whether a pipeline strategy is successful or not.
GlaxoSmithKline ($GSK) faced that bitter truth after its batch of NDAs failed to deliver sufficient product sales to prevent a reorganization of its R&D division this year. Now AstraZeneca ($AZN) has to demonstrate if it can deliver and capitalize on new approvals--as CEO Pascal Soriot promised so elaborately in 2014--or leave investors disappointed by hard numbers. And Amgen ($AMGN) faces exactly the same task.
The flip side to more innovation, adds Munos, is more competition. "Viekira Pak is showing what that means for pricing," he writes. "It's not an isolated example. Lilly has cautioned that diabetes drug sales--the company's bright spot--are expected to slow next year due to new pricing pressure in the U.S. FiercePharma has identified eight therapeutic areas where price competition is likely to intensify. I would add asthma to the list. It means that companies will soon have to deal with a challenge they've tried to ignore, i.e., the enormous cost of their legacy structures. As a result of patent cliffs, Big Pharma has become big generic. Forty one percent ($123 billion) of the product sales ($300 billion) reported by Big Pharma in 2013 are no longer protected by patents. It still pays a lot of bills, but it's eroding, and it's not a good business. Someone must deal with this. The leaders will, but not every company has them."
Gilead (Harvoni and Zydelig), Bristol-Myers Squibb (Opdivo), Celgene (Otezla) and Biogen Idec (Plegridy), meanwhile, continued to prove how dominant biotechs with carefully focused pipelines can deliver major new approvals with significantly less spent on R&D.
For the record, Munos keeps a different count for what should be on the innovative new drug approvals for 2014. He adds Biogen's Alprolix and Eloctate, two hemophilia drugs that were licensed in, along with Baxter's Obizur for hemophilia, Salix's Ruconest as well as Trumenba, an rDNA vaccine to protect against Neisseria meningitidis Type B, for which Pfizer received a Breakthrough Therapy Designation. But he also subtracts the two imaging agents that are on the FDA's list. -- John Carroll (email | Twitter)
January
| 1. Farxiga | |
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Active ingredient: dapagliflozin Disease: diabetes Peak sales estimate: $1.5 billion Approved: Jan. 8 Company: AstraZeneca |
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The Scoop: Long-delayed SGLT-2 drug Farxiga, advanced by Bristol-Myers Squibb ($BMY) and AstraZeneca ($AZN) as part of their big diabetes partnership in the wake of the $7 billion Amylin buyout, finally won the FDA's blessing in January. But since then, the med--which flushes glucose in urine to help diabetics achieve glycemic control--has gained two new in-class rivals in Johnson & Johnson's ($JNJ) Invokana and Eli Lilly ($LLY) and Boehringer Ingelheim's Jardiance. And they don't come with the bladder cancer worries the FDA has flagged with Farxiga. -- Carly Helfand
For more:
AstraZeneca wins another diabetes approval in a fast-crowding market
AZ, BMS create 'Diabetes Inc.,' combine marketing in new home
Bristol, AstraZeneca chalk up EU approval for Forxiga after FDA rejection
| 2. Hetlioz | |
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Active ingredient: tasimelteon Disease: non-24-hour sleep-wake disorder Peak sales estimate: $600 million Approved: Jan. 31 Company: Vanda Pharmaceuticals |
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The Scoop: A lot is riding on the success of Vanda's orphan drug Hetlioz, given that the company's only other product, Fanapt, never gained traction in the schizophrenia space. Hetlioz was enthusiastically endorsed by FDA reviewers who said its ability to safely reset the circadian rhythms of the blind significantly improved their sleep--more nighttime sleep, less daytime napping. It is the first drug approved for non-24 in the U.S., and there are about 80,000 potential users. As an orphan, Vanda was able to tag with a premium price, $75,000 after rebates and discounts, according to a note from Morningstar analyst Stefan Quenneville. Vanda's difficulty will be attaining the drug's sales potential given the company's relatively small sales infrastructure. A direct-to-consumer sales campaign has gone a long way to building sales, $5.2 million, up 30%, in its second quarter on the market. But that came at a significant cost, Quenneville pointed out, with its sales, general and administrative costs topping $28 million during the first quarter of its launch. The company does not expect to rely on just one market or one indication. It has an application for Hetlioz pending in the EU, and Vanda is working on an observational study in patients with the rare sleep disorder Smith-Magenis syndrome. -- Eric Palmer
For more:
Special Report: Pharma's Biggest Flops - Fanapt
Vanda jumps again as FDA panel smiles on sleep-disorder drug
Vanda soars after FDA reviewer urges OK for sleep disorder drug
Vanda reaps $200M upfront in Novartis drug pact
February
| 3. Vimizim | |
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Active ingredient: elosulfase alfa Disease: mucopolysaccharidosis type IVA (Morquio A syndrome) Peak sales estimate: $628.4 million Approved: Feb. 14 Company: BioMarin Pharmaceuticals |
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The Scoop: Vimizim treats the extremely rare enzyme deficiency, Morquio A, and getting FDA approval was a piece of cake for BioMarin ($BMRN). The condition, which is estimated to affect 3,000 patients in the developed world and only 800 in the U.S., causes joint abnormalities, heart problems and hearing loss, as well as other bone problems. A committee of FDA advisers voted 20 to 1 in favor of approval. Despite the low patient population, Vimizim has the potential to hit hundreds of millions in sales, thanks to its high price tag, $380,000 a year. -- Amirah Al Idrus
For more:
BioMarin's $380K price tag on Vimizim is high, but far from the highest
BioMarin skates through FDA panel for rare disease drug
BioMarin and its patient coaches are ready to roll on Vimizim launch
Vimizim panel nod fuels new BioMarin buyout buzz
| 4. Northera | |
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Active ingredient: droxidopa Disease: neurogenic orthostatic hypotension Peak sales estimate: $300 million to $450 million Approved: Feb. 18 Company: Chelsea Therapeutics |
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The Scoop: Chelsea Therapeutics' Northera is the first drug in more than two decades approved for treating neurogenic orthostatic hypotension. NOH is a rare, but chronic, and sometimes debilitating, sudden fall in blood pressure that afflicts some people with Parkinson's disease and some other conditions. But it will be a tricky sale, given the caveats that come with it. It has a black-box warning for stroke risk because it raises people's blood pressure while they're lying down, so the FDA says patients should sleep with their upper body elevated. And initial trials were only for two weeks of treatment, so it isn't demonstrated it will be effective after that. All of that adds up to the kind of challenge that Denmark's Lundbeck thought it was equipped to address. It bought out Chelsea and Northera in a deal that may eventually add up to $658 million. Lundbeck hopes the drug can be at least part of the answer to its anemic financials. Its earnings hit the skids with the 2012 patent loss of Lexapro, the blockbuster antidepressant it sold with Forest Laboratories ($FRX). It says its experience with rare and challenging neurological disorders make it "uniquely" positioned to get the most from the treatment. Besides Lexapro, it has sold the antidepressant Avapro and since its 2013 approval in Europe has been marketing its anti-binge drinking drug Selincro. -- Eric Palmer
For more:
Lundbeck makes $658M bid for Chelsea, gaining once-rejected dizziness drug Northera
Chelsea wins FDA nod for once-spurned Northera, but success isn't guaranteed
Chelsea soars after FDA AdCom once again endorses bid to OK Northera
FDA reviewer flunks Chelsea's effort at a comeback for Northera
| 5. Myalept | |
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Active ingredient: metreleptin for injection Disease: generalized lipodystrophy Peak sales estimate: $300 million Approved: Feb. 24 Company: AstraZeneca |
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The Scoop: Myalept has an interesting chain of ownership. It was initially developed by San Diego-based Amylin as an obesity treatment. Amylin was bought by the Bristol-Myers Squibb ($BMY) and AstraZeneca ($AZN) diabetes partnership in 2012 for $7 billion. The next year, AstraZeneca ended up with the drug after buying out BMS' share of their joint operation. AstraZeneca had sought approval of metreleptin for treating patients with metabolic disorders associated with partial lipodystrophy, a potentially fatal condition that affects a few thousand people in the world. It was never going to be a big seller, maybe $300 million at its peak, analysts forecast, but the FDA only approved it for a subset of that group, generalized lipodystrophy, further compressing its sales potential. On top of that, the FDA ordered 7 postmarketing studies and requested 8 more. In November, AstraZeneca rid itself of the drug, selling it to Aegerion Pharmaceuticals ($AEGR) for $325 million up front for the global rights, except in Japan, South Korea, and Taiwan, where Shionogi has rights to it. Aegerion said it is well positioned to make the most of the drug, given its experience with rare disease treatments. Aegerion's only other product is Juxtapid, a pill approved in late 2012 to lower cholesterol levels in patients with a rare genetic disease, familial homozygous hypercholesterolemia. -- Eric Palmer
For more:
FDA OKs AstraZeneca's rare disease drug, but only for a subset of patients
FDA approves Aegerion's lomitapide for rare cases of HoFH
FDA panel throws dirt on Bristol-Myers' orphan drug metreleptin
AstraZeneca will pay up to $4.1B for Bristol's share of their diabetes pact
FDA raises safety, efficacy questions for Bristol-Myers' rare-disease drug metreleptin
March
| 6. Neuraceq | |
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Active ingredient: florbetaben F 18 injection Disease: Alzheimer's disease Peak sales estimate: n/a Approved: March 19 Company: Piramal Imaging |
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The Scoop: The market for amyloid beta imaging agents designed to diagnose neurodegenerative diseases such as Alzheimer's has had some trouble getting off the ground. Uncertainty as to their effectiveness has led to regulatory caution, and a lack of successful Alzheimer's drugs has hindered the diagnostics' progress. But several agents have garnered limited approval from the FDA, including GE Healthcare's ($GE) Vizamyl, Eli Lilly's ($LLY) Amyvid and, new this year, Piramal Imaging's Neuraceq. The amyloid beta PET tracer is designed to detect plaque density in the brains of patients with cognitive impairment. But these tests, according to the FDA, can't determine with certainty whether the impairment is due specifically to Alzheimer's--hence, the limited review. This kind of early diagnosis, though, is still important in the Alzheimer's arena and is another step forward in a difficult biotech sector. Back in 2012, Piramal bought Bayer's portfolio of molecular imaging agents in a market environment that wasn't too different than it is now with regards to clinical and regulatory uncertainty. But back then, they claimed that the market for such markers could amount to $1.5 billion, an amount that has only become greater. -- Michael Gibney
For more:
AC Immune and Piramal Imaging link in Alzheimer's imaging agent development deal
Vizamyl: The FDA OKs an imaging product without a market
| 7. Impavido | |
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Active ingredient: miltefosine Disease: leishmaniasis Peak sales estimate: n/a Approved: March 19 Company: Knight Therapeutics |
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The Scoop: Montreal-based Knight Therapeutics spun off from Paladin Therapeutics after Endo ($ENDP) bought Paladin. Knight took along with it one compound: miltefosine, known as Impavido, a treatment for the rare tropical disease leishmaniasis. Impavido had been approved throughout the world and brought in, according to a report from CanTech Letter, about $2.5 million annually at the time. And Knight's main goal, it seemed, was to collect a highly valuable priority review voucher (PRV) from the FDA, which it did in March when the U.S. agency approved Impavido there. That PRV is transferable, and that's just what Knight did, selling the voucher to Gilead ($GILD) for a record sum of $125 million in November, giving the aggressive developer a regulatory shortcut for a 6-month review. How and when Gilead will be using the voucher is still up in the air, but the record sale showed just how valuable these vouchers can be, a strategy Knight had in mind. "We are thrilled to successfully transfer our priority review voucher in expectation that this will encourage others to invest in R&D for neglected tropical diseases for the benefit of humanity," said Jonathan Ross Goodman, director of Knight. -- Michael Gibney
For more:
Go-go execs at Gilead nab an FDA priority review voucher for $125M
| 8. Otezla | |
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Active ingredient: apremalist Disease: active psoriatic arthritis Peak sales estimate: $1.2 billion consensus Approved: March 21 Company: Celgene |
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The Scoop: Celgene's ($CELG) regulatory win in March for psoriatic arthritis was a prelude to its even bigger OK for psoriasis, the most common autoimmune disease in the U.S. And it's a pioneer, the first in a new class of drugs that have been in development, and Eli Lilly ($LLY), Novartis ($NVS), Amgen ($AMGN), AstraZeneca ($AZN), Merck ($MRK) and Johnson & Johnson ($JNJ) are all hustling up from behind to stake their own claims in this blockbuster field. Celgene, though, has high hopes that its oral drug can start to carve away sales from the likes of Humira, a megablockbuster that has dominated the market. Enrel and Remicade are two other mainstays likely to face considerable pressure, especially as biosimilars makers come along with copycat versions of these drugs. Celgene managed to get there first, and it will do everything it can to make the most of first-mover status. -- John Carroll
For more:
Celgene wins FDA approval for its blockbuster arthritis contender apremilast
Celgene's would-be blockbuster fails in Phase III for spinal arthritis
Celgene bolsters case for closely watched arthritis drug with Ph3 data
Celgene nabs another round of promising pivotal apremalist data for psoriatic arthritis
April
| 9. Tanzeum | |
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Active ingredient: albiglutide Disease: diabetes Peak sales estimate: $218 million Approved: April 15 Company: GlaxoSmithKline |
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The Scoop: Glaxo ($GSK) joined the diabetes fray with the U.S. approval of its GLP-1 agonist Tanzeum (albiglutide), a once-weekly treatment designed to boost insulin production in patients with Type 2 diabetes. In April, Tanzeum was the newest entry into the GLP-1 market, which analysts at the time predicted could reach a hefty $5 billion a year. But Glaxo's drug entered the arena facing some much heavier hitters, including market leader Victoza from Novo Nordisk ($NVO) and Bydureon from AstraZeneca ($AZN). And following close behind this year was Eli Lilly's ($LLY) Trulicity (dulaglutide) with an approval of its own in September and a peak sales estimate about 6 times superior to Tanzeum's. Lilly's also boasts a once-weekly dose, giving Tanzeum little else to stand apart. Sanofi ($SNY) is also looking to get in on the GLP-1 action down the road with its lixasenatide, a treatment that won't be approved until at least 2016 but that has analysts looking at sales of $450 million, still more than Glaxo's. -- Michael Gibney
For more:
GlaxoSmithKline wins an FDA nod for its GLP-1 diabetes drug albiglutide
Newly minted GlaxoSmithKline diabetes drug faces formidable foes in GLP-1 market
| 10. Cyramza | |
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Active ingredient: ramucirumab Disease: advanced stomach cancer Peak sales estimate: $1.3 billion Approved: April 21 Company: Eli Lilly |
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The Scoop: Cyramza has an outsized role to play at Eli Lilly ($LLY). Its first approval for stomach cancer marked a major step for a company that has experienced a long dry spell in the clinic. And it marked a long-awaited R&D payback for its $6.5 billion ImClone buyout way back in 2008. The drug, though, has a weak set of data to back it up in stomach cancer and a hit-and-miss record in the clinic as Eli Lilly pushes hard to make this drug into a blockbuster. It's come through in colorectal cancer and looks marginal in lung cancer, setting it up as a likely second-line therapy. It's also failed for liver cancer and breast cancer. With revenue plummeting during what is widely perceived as a trough year after key franchises lost patent protection, making this drug work on the market is essential. But Lilly hasn't been anxious to discuss early sales figures. The heat is on. -- John Carroll
For more:
Lilly's blockbuster hopeful Cyramza nabs key new use in lung cancer
Lilly says with new data, it will seek another approval for Cyramza next year
In another stinging setback, Eli Lilly's ramucirumab fails PhIII breast cancer study
| 11. Sylvant | |
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Active ingredient: siltuximab Disease: multicentric Castleman disease Peak sales estimate: n/a Approved: April 23 Company: Johnson & Johnson |
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The Scoop: Sylvant became the first drug approved to treat patients with the rare disease multicentric Castleman's disease, which is characterized by abnormal growth of immune cells in the lymph nodes. The drug has a patient population of only 1,200 people. MCD is similar to lymphoma, but not classified as a cancer, says the American Cancer Society. The drug triggered a tumor response in about a thirds of its patients during clinical trials. "MCD is a complex disease and up until this point, physicians have tried to reduce lymph node masses and put the disease in remission through a combination of treatments, but MCD often returns. Today's approval of Sylvant gives physicians a long-awaited treatment option for a group of patients who has been suffering with this chronic, serious and debilitating disease," said the J&J ($JNJ) lead investigator, Frits van Rhee, in a statement. At the time of approval, the active ingredient, an anti-IL-6 antibody that's delivered in an IV formulation, was being studied for multiple myeloma, but many those of studies have since been withdrawn or terminated due to a lack of efficacy, according to ClinicalTrials.gov. -- Varun Saxena
For more:
J&J snags FDA OK for rare blood disorder drug siltuximab
| 12. Zykadia | |
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Active ingredient: ceritinib Disease: lung cancer Peak sales estimate: $330 million by 2018 Approved: April 29 Company: Novartis |
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The Scoop: The "breakthrough" approval of Zykadia after just three years of clinical testing was hailed as a sign of the FDA's willingness to speed promising therapies to the market. FDA cancer drug czar Richard Pazdur said that the approval "demonstrates the FDA's commitment to working cooperatively with companies to expedite a drug's development, review and approval, reflecting the promise of the breakthrough therapy designation program." Zykadia was approved as a second-line therapy for for ALK-positive non-small cell lung cancer, following treatment with Pfizer's ($PFE) Xalkori (crizotinib), meaning it will be difficult to achieve the coveted title of "blockbuster drug." Xalkori comes with a companion diagnostic to test for a defect in the Alk gene, to the benefit of Novartis ($NVS), which did invest in a test of its own due to Zykadia's second-line status. That leaves some people wondering why at $13,200 per month, the drug is more expensive than Pfizer's option. In a 114-person clinical trial, Zykadia achieved a 58% response rate and a median progression-free survival rate of 7 months. -- Varun Saxena
For more:
Novartis grabs early FDA approval of lung cancer drug Zykadia
The irrational rationale for cancer drug prices, with Novartis' Zykadia as exhibit A
May
| 13. Zontivity | |
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Active ingredient: vorapaxar Disease: heart and peripheral vascular disease Peak sales estimate: initially $5 billion, now much, much less Approved: May 8 Company: Merck |
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The Scoop: Merck's ($MRK) Vorapaxar is a potential replacement for the popular blood thinner warfarin but comes with a black-box warning for excessive bleeding. Safety concerns mean vorapaxar is contraindicated for patients who have already had strokes or transient ischemic attack, and the initial peak sales estimate of $5 billion is overoptimistic. Still, the first-in-class inhibitor of the PAR-1 receptor is FDA-approved "to reduce the risk of heart attack, stroke, cardiovascular death, and need for procedures to restore the blood flow to the heart in patients with a previous heart attack or blockages in the arteries to the legs." That's a market size of at least 7.6 million Americans, or the number of people in the U.S. who have suffered a heart attack. In clinical trials, Vorapaxar reduced the risk of a stroke from 9.5% to 7.9% over three years. The then-candidate was one of the reasons behind Merck's acquisition of Schering-Plough in 2009. -- Varun Saxena
For more:
Merck wins FDA approval for a once-vaunted cardio drug
Merck clears an FDA panel with its clot-fighting vorapaxar
| 14. Entyvio | |
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Active ingredient: vedolizumab Disease: ulcerative colitis and Crohn's disease Peak sales estimate: $1 billion Approved: May 20 Company: Takeda |
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The Scoop: Takeda hopes it has a blockbuster with Entyvio despite the fact that it isn't for first-line use amidst a very crowded field of competitors. The drug had some setbacks on its primary and secondary endpoints and progressive multifocal leukoencephalopathy has been identified as a potential safety issue, given the drug's similarity to Biogen Idec's ($BIIB) Tysabri. Both drugs are integrin receptor antagonists. In the Entyvio panel, most members found safety sufficient when used in combination with immunosuppressants. But the panel split over whether or not the drug should be used as front-line induction therapy; the FDA opted for a more cautious second-line approach. Analyst Tim Race of Deutsche Bank expects Entyvio will hit $1.06 billion in peak sales in 2020, with 80% of that in ulcerative colitis and the remainder in Crohn's disease. If it does move on that trajectory, Entyvio would contribute more than 10% of Takeda's core earnings in 2018. -- Stacy Lawrence
For more:
Takeda sways FDA panel with its bowel disease drug despite viral risks
Takeda overcomes safety questions, wins FDA OK for IBD drug vedolizumab
Second-line use, crowded market won't keep Takeda's Entyvio from blockbuster run
| 15. Dalvance | |
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Active ingredient: dalbavancin Disease: acute bacterial skin and skin structure infections (ABSSSI) Peak sales estimate: $400 million Approved: May 23 Company: Actavis |
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The Scoop: Dalvance was the very first drug designated as a Qualified Infectious Disease Product to receive FDA approval. That designation was introduced as part of the Generating Antibiotic Incentives Now (GAIN) legislation in the FDA Safety and Innovation Act that was signed into law in July 2012. The idea was to encourage the development of new antibiotics to combat increasing antibiotic drug resistance. The Dalvance approval was sufficiently compelling that it sparked a $675 million buyout, plus a $5 per share contingent value right based on new indications for the IV antibiotic, by Actavis ($ACT) of Durata Therapeutics that closed in November. Additional indications could include hospitalized community-acquired pneumonia and pediatric osteomyelitis; a supplemental NDA is planned in mid-2015. The drug has a long and storied history. It was out-licensed by Durata from Pfizer ($PFE) after FDA had rejected dalbavancin in 2007. FDA was in the midst of changing its guidance for the clinical testing it required for antibiotics; Durata based its pivotal testing on draft guidance issued by FDA in 2010 for the indication of ABSSSI. Durata had an IPO in July 2012; its pre-IPO investors included Domain Associates, New Leaf Ventures, Aisling Capital, Sofinnova Venture Partners, Canaan Partners and Quaker BioVentures. -- Stacy Lawrence
For more:
Actavis snaps up Durata, newly approved antibiotic in $675M buyout
Faded star at Pfizer leads to discounted $68M IPO for Durata
Little Durata's relocation plans reap incentives with big staffing promises
June
| 16. Jublia | |
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Active ingredient: efinaconazole 10% topical solution Disease: onychomycosis of the toenails Peak sales estimate: $300 million to $800 million in the U.S. Approved: June 9 Company: Valeant Pharmaceuticals |
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The Scoop: Valeant ($VRX) has built a massive empire by wrapping up a seemingly unending stream of small, marketed specialty products into its massive portfolio. But this time around, Valeant could have a winner that it's created almost entirely on its own--that is expected to ultimately become its largest product. Valeant acquired Jublia when it bought Dow Pharmaceutical Sciences in 2008. But the candidate was still preclinical then. The M&A specialist managed to get Jublia through the R&D process successfully--and now the drug may prove to be its top seller if it hits company and analyst estimates. This is the fourth product approval that Valeant has generated from those Dow assets. Jublia was rejected last year by FDA due to issues with its container, but the company successfully addressed its concerns. Already after a July launch in the U.S. and Canada, Jublia was Valeant's 28th largest product during the third quarter. It expects Jublia will be a top 20 product by sales during the fourth quarter. No product accounts for more than 3.5% of total Valeant revenues, which are expected to come in at $8.1 to $8.3 billion during 2014. The company has started what it expects will be a massive direct-to-consumer outreach. The company said it expects more than $150 million in Jublia revenues in 2015, with about $300 million in 2016. "We continue to believe that Jublia will likely become our largest product," reaffirmed Valeant chairman and CEO J. Michael Pearson on the third quarter earnings call. -- Stacy Lawrence
For more:
Valeant ordered to pay Anacor $100M
FDA manufacturing concerns hold up Valeant drug launch
| 17. Sivextro | |
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Active ingredient: tedizolid phosphate Disease: skin infections in adults Peak sales estimate: $300 million Approved: June 20 Company: Cubist Pharmaceuticals |
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The Scoop: Not even a year after Cubist ($CBST) acquired Trius in an effort to grow its presence in antibiotics, it scooped up an FDA approval for Sivextro. The antibacterial drug, another weapon in the war against antibiotic-resistant bacteria, is approved in tablet and injection form for the treatment of a range of skin infections, including methicillin-resistant Staphylococcus aureus. It became a direct competitor to Pfizer's ($PFE) Zyvox. In clinical trials, it performed as well as Zyvox, but with a shorter treatment time, less frequent dosing and fewer side effects. Zyvox made $1.4 billion in sales last year, and Sivextro could be hot on its heels. -- Amirah Al Idrus
For more:
Cubist's Trius buyout pays off with FDA nod on high-powered antibiotic Sivextro
Cubist's superbug drug clears FDA panel, lines up for Pfizer showdown
Cubist splurges on Trius and Optimer buyouts to spur antibiotics growth
Trius targets key approvals after antibiotic matches Zyvox (again) in Phase III
July
| 18. Beleodaq | |
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Active ingredient: belinostat Disease: peripheral T-cell lymphoma (PTCL) Peak sales estimate: $130 million Approved: July 3 Company: Spectrum Pharmaceuticals |
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The Scoop: Beleodaq is the third drug to be approved for the treatment of PTCL--a rare and fast-growing form of non-Hodgkin lymphoma--since 2009. It joins Folotyn, also marketed by Spectrum, in 2009, and Istodax, marketed by Celgene ($CELG), in 2011. PTCL is a group of diseases that are characterized by cancerous lymph nodes. Beleodaq works by blocking enzymes that contribute to T cells becoming cancerous. It is intended for patients who have relapsed, or who did not respond to previous treatment. Beleodaq was approved under FDA's accelerated approval program and also received orphan drug designation. -- Amirah Al Idrus
For more:
FDA gives an early OK to lymphoma drug Beleodaq Spectrum touts PhII cancer data, plans NDA
Spectrum touts PhII cancer data, plans NDA
Spectrum seeks rapid approval for anticancer contender
| 19. Kerydin | |
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Active ingredient: tavaborole Disease: onychomycosis of the toenails Peak sales estimate: $600 million Approved: July 7 Company: Anacor Pharmaceuticals |
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The Scoop: Approximately 35 million people in the U.S. have onychomycosis in their toenails, but only 5 to 6 million of them have been diagnosed with the pesky fungus. And of those diagnosed with the difficult-to-treat, recurring fungal infection, a large number of them has historically chosen not to treat the infection. Enter Kerydin, the first oxaborole antifungal agent developed for topical treatment of onychomycosis of the toenails. The clear, colorless, alcohol-based solution is applied to the toes with a dropper, once daily for 48 weeks. It joins Jublia, approved a month prior, which is an efinaconazole-based topical remedy for the fungus. -- Amirah Al Idrus
For more:
Anacor Pharmaceuticals and Sandoz Enter Into an Agreement for the Commercialization of KERYDIN in the United States
FDA Approves Anacor Pharmaceuticals' KERYDIN™ (Tavaborole) Topical Solution, 5% for the Treatment of Onychomycosis of the Toenails
| 20. Zydelig | |
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Active ingredient: idelalisib Disease: B-cell blood cancers (chronic lymphocytic leukemia, relapsed follicular B-cell non-Hodgkin lymphoma and small lymphocytic lymphoma) Peak sales estimate: $1.5 billion in annual sales by 2017 Approved: July 23 Company: Gilead Sciences |
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The Scoop: It has been a blockbuster year for Gilead Sciences ($GILD), and the company added another notch to its belt in July when it won the FDA's blessings for its PI3k inhibitor Zydelig. Approved for three types of B-cell blood cancers, the drug bested Roche's ($RHHBY) Rituxan in late-stage trials, scoring a higher progression-free survival rate when paired with Rituxan and better overall response rates than Roche's drug on its own. But Zydelig also faces competition from Johnson & Johnson's Imbruvica and AbbVie's ($ABBV) Rituxan add-on ABT-199, not to mention the bane of an FDA black-box warning that highlights serious risks, including potentially fatal liver problems. Still, analysts figure that Gilead will end up snatching up 10% to 20% of the market with Zydelig, and that the drug will hit eye-popping sales of $1.2 billion by 2020. -- Emily Wasserman
For more:
Gilead picks up blockbuster FDA blood cancer approvals for idelalisib
Can Gilead break into cancer? With Zydelig approval, it gets its chance
Gilead's Zydelig, J&J's Imbruvica will go toe-to-toe in EU with nod for CLL
Gilead scores early PhIII success for idelalisib in chronic lymphocytic leukemia
August
| 21. Striverdi Respimat | |
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Active ingredient: olodaterol Disease: chronic obstructive pulmonary disease (COPD) Peak sales estimate: n/a Approved: Aug. 1 Company: Boehringer Ingelheim |
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The Scoop: The respiratory market is in the midst of a shake-up, with older blockbusters becoming vulnerable to generic competition and new entrants hoping to pick up share. Boehringer now has both: its Spiriva franchise, a long-acting muscarinic antagonist (LAMA) worth $4.7 billion, and its brand-new Striverdi, a long-acting beta agonist (LABA) delivered via its Respimat device. As a single-agent COPD remedy, Striverdi has its work cut out for it, considering the copycat versions of GlaxoSmithKline's ($GSK) Advair and AstraZeneca's ($AZN) Symbicort due on the scene. And with a variety of combination LABA/LAMA drugs coming down the pike, Boehringer's respiratory business won't pump up on Striverdi alone. Two weeks after winning approval for that drug, the German pharma asked FDA to approve its follow-up combo treatment, which puts Striverdi into the same package with Spiriva. Citigroup expects the COPD market to swell to $14 billion by 2018, from $10 billion this year, and Boehringer's stable will be jockeying with AstraZeneca's Duaklir, Glaxo's Anoro and Breo, and Novartis' ($NVS) Seebri, Onbrez and a combo of the two now known as QVA149. -- Tracy Staton
For more:
Can Boehringer carve out space in a crowded COPD market with new approval?
FDA experts endorse new COPD blockbuster from GlaxoSmithKline, Theravance
U.K., Denmark, Iceland all go for Boehringer Ingelheim COPD drug Striverdi Respimat
AstraZeneca's Almirall buy yields its first new drug--and now, AZ has to sell it
| 22. Jardiance | |
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Active ingredient: empagliflozin Disease: diabetes Peak sales estimate: about $980 million by 2020 Approved: Aug. 1 Company: Eli Lilly/Boehringer Ingelheim |
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The Scoop: Jardiance had already had one FDA slap-down in the face of a manufacturing issue when the agency came around and gave it a go-ahead in August. And since then, Eli Lilly ($LLY) and Boehringer Ingelheim's med has had to go up against fellow SGLT-2 inhibitors Invokana from Johnson & Johnson ($JNJ) and Farxiga from AstraZeneca ($AZN). But Jardiance has something those competitors don't have: A clean safety slate. The FDA has flagged Farxiga over bladder cancer worries, and Invokana does not have a nod for patients with severe liver damage. But as it did with Jardiance's rivals, the agency required it to undergo a cardiovascular outcomes trial and some pediatric studies to affirm its tolerability. -- Carly Helfand
For more:
Third to market may still be a charm for Lilly, BI's new diabetes med Jardiance
FDA comes around on Boehringer, Lilly's diabetes med Jardiance
FDA-nixed diabetes drug from Lilly, Boehringer could face rough path to stardom
| 23. Orbactiv | |
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Active ingredient: oritavancin Disease: acute bacterial skin infections caused by Staphylococcus aureus, Streptococcus and Enterococcus Peak sales estimate: $450 million Approved: Aug. 7 Company: The Medicines Company |
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The Scoop: Patent-cliff pain isn't limited to big drugmakers. The Medicines Co., a sub-$1 billion pharma company, has been working for years to prepare for generic competition for the clot-fighter Angiomax, its top-selling product. In 2009, the company picked up Targanta Therapeutics for $42 million plus potential milestones, eyeing oritavancin and its ability to fight "superbug" infections. When Medicines Co. lost a court fight with Hospira in March, opening the door to earlier-than-expected generic rivals to Angiomax, that prep work grew more important. And after the FDA rejected its experimental clot-fighting follow-up, Cangrelor, in May, Orbactiv itself became more important than ever. The drug treats serious and complex skin infections, including those caused by drug-resistant S. aureus, a growing problem in hospitals in the U.S. and Europe. Orbactiv has a couple of other new antibiotics to contend with, including Cubist's ($CBST) Sivextro, approved in June. But as a one-dose-only drug, Orbactiv may have an advantage. The company itself sees peak sales at $450 million, though analysts are bit less optimistic; Guggenheim's Louise Chen figures on $200 million by 2021. -- Tracy Staton
For more:
After a 6-year delay, Medicines Co. bags FDA approval for once-troubled antibiotic
Medicines Company sues lawyers for busting Angiomax patent filing deadline
Medicines Co. snags Targanta for $42M-plus
When will Big Pharma get serious about antibiotic R&D?
Merck adds heft to hospital biz with $9.5B Cubist buyout
| 24. Belsomra | |
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Active ingredient: suvorexant Disease: insomnia Peak sales estimate: $500 million Approved: Aug. 14 Company: Merck |
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The Scoop: Merck ($MRK) made a big play early on to hype the potential of this sleep drug, excited that it was working with a new mechanism of action. But by the time the FDA was finished with this drug, which block the brain's orexin receptors, the pharma giant had toned down its forecasts considerably. To be fair, investigators were developing this drug on a changing playing field. As regulators grew more and more alarmed by the damage done with existing therapies, they began to force down dosages. Merck's drug was approved at a dosage the company's own team had publicly disavowed as ineffective. And in the face of a big group of cheap generics on the market, analysts gradually whittled down some grandiose peak sales estimates. The focus at Merck now, mercifully, is on immuno-oncology and hep C. -- John Carroll
For more:
Merck's sleep drug suvorexant overcomes hurdles to win FDA approval
Merck wins FDA nod for a weaker form of its embattled sleep drug
FDA scraps Merck's high-dose sleep drug, demands weaker version
FDA panel backs low-dose rollout of Merck's sleep drug suvorexant
| 25. Plegridy | |
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Active ingredient: peginterferon beta-1a Disease: Multiple sclerosis Peak sales estimate: $1 billion by 2027 Approved: Aug. 15 Company: Biogen Idec |
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The Scoop: Tecfidera is the multiple sclerosis drug making most of the headlines for Biogen Idec ($BIIB) these days, what with its convenient oral dosing and zero-to-blockbuster sales. But before there was Tecfidera, Biogen had Avonex, and the beta interferon treatment has long been a solid performer for the Cambridge, MA-based company. With Avonex nearing the end of its patent life, Biogen developed a long-acting version, dosed every two weeks rather than once a week. And that's Plegridy. It's not expected to deliver the revenue bang of a Tecfidera, but analysts do see Plegridy upholding the franchise. They predict it will pick up sales where Avonex leaves off, delivering $1 billion in annual revenue by 2027. Biogen itself believes that shorter-acting interferon meds, such as Merck Serono's Rebif, injected three times a week, are vulnerable to competition from a longer-acting option. The company hopes its specialized multiple sclerosis sales force--experienced with Avonex, Tecfidera and Tysabri--can persuade doctors to give it a try. -- Tracy Staton
For more:
Special Report: The top 10 best-selling multiple sclerosis drugs of 2013
Biogen ready to steal market share with newly minted MS drug Plegridy
Biogen scores fresh MS advance with blockbuster Plegridy OK
Biogen Idec advances blockbuster MS franchise on two key fronts
| 26. Cerdelga | |
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Active ingredient: eliglustat Disease: Gaucher's disease Peak sales estimate: up to $750 million Approved: Aug. 19 Company: Sanofi/Genzyme |
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The Scoop: Following its own trailblazing work in Gaucher's disease with the intravenous Cerezyme, Sanofi's ($SNY) Genzyme unit capped 15 years of R&D work with Cerdelga, an oral treatment for the rare ailment. The drug, developed as eliglustat, is designed to treat the symptoms of Gaucher's, which stems from a deficiency of the enzyme glucocerebrosidase and leads to fatty buildups in the spleen and liver. Unlike Cerezyme, which replaces the missing component, the new drug is designed to inhibit the metabolic process that helps the deposits form, eschewing injections in favor of a twice-a-day pill. That could make a big difference for many of the roughly 10,000 Gaucher's patients around the world, according to Genzyme, but the company may find itself in a zero-sum sales game, as a sizable chunk of Cerdelga's market opportunity will come at Cerezyme's expense. -- Damian Garde
For more:
Genzyme puts $310,250 price tag on new Gaucher-fighting pill
Sanofi wins FDA's blessing for an oral Gaucher drug
Sanofi reports positive PhIII data on oral Gaucher drug
September
| 27. Keytruda | |
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Active ingredient: pembrolizumab Disease: melanoma Peak sales estimate: $4.4 billion Approved: Sept. 4 Company: Merck |
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The Scoop: It was an intense race to the top of the PD-1 blockbuster heap, but Merck ($MRK) emerged a victor when the FDA in September signed off on its skin cancer treatment, Keytruda. The product spurs an immune system attack on tumors by blocking a pathway called PD-1, which normally allows cancerous cells to pass through undetected, and is a beacon of hope for Merck as it ramps up its immuno-oncology program and hits the reset button on R&D. The company is also pushing for multiple applications for Keytruda--and is chalking up study data to make its case. Merck at September's European Society for Medical Oncology meeting said Keytruda delivered a 24% response rate in patients with bladder cancer when used as a standalone treatment, and 10% of patients saw a complete response. -- Emily Wasserman
For more:
Merck grabs another 'breakthrough' in PD-1 lung cancer race with Bristol-Myers
Yervoy, Sprycel, Eliquis jumps help Bristol-Myers beat Q3 estimates
ESMO: Bristol-Myers, Roche, AstraZeneca, GSK, Merck jostle for spotlight
Bristol-Myers socks Merck's brand-new Keytruda with PD-1 patent suit
Merck wins breakthrough FDA approval for blockbuster cancer contender pembrolizumab
| 28. Movantik | |
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Active ingredient: naloxegol Disease: opioid-induced constipation in adults with chronic noncancer pain Peak sales estimate: $1 billion-plus Approved: Sept. 16 Company: AstraZeneca, Nektar |
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The Scoop: AstraZeneca ($AZN) and its partner Nektar had the distinction of being the first to gain an approval for a new mu opioid drug specifically OK'd for opioid-induced constipation, and the pharma giant is absolutely determined to make it a blockbuster. First, it has to clear the DEA, but once on the market it appears well positioned to grab market share. Salix managed to gain an approval for the same indication with Relistor, but it's an injectable and Movantik is an oral drug. There are also rivals from Sucampo and Cubist to consider. On the down side, though, the partners also have to deal with evidence that the drug is linked to heart attacks, forcing a large postmarketing study on the risks it presents. AstraZeneca inked a $1.5 billion deal for this drug in 2009. These days, it's running hard to dispel its rep as a laggard in the industry. And Movantik will be a key exhibit as the company makes its case. -- John Carroll
For more:
AstraZeneca and Nektar win FDA OK for blockbuster-hopeful constipation drug
AZ faces market-making task to capitalize on Movantik's edge in OIC
FDA panel advises against more clinical trials for opioid constipation drugs
AstraZeneca claims PhIII sweep for OIC drug as CV concerns linger
| 29. Trulicity | |
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Active ingredient: dulaglutide Disease: diabetes Peak sales estimate: ranges up to $1.2 billion Approved: Sept. 18 Company: Eli Lilly |
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The Scoop: Eli Lilly ($LLY) promised investors that it would end its long dry spell on the NDA front with at least three new approvals this year, but it's Trulicity that promises to stand out of the small pack with substantial revenue. Lilly tailored its 9-part Phase III program to pit dulaglutide against its most likely competitors, and the drug has yet to disappoint. Lilly's once-weekly treatment has thus far bested AstraZeneca's ($AZN) Byetta, Merck's ($MRK) Januvia and metformin in controlling blood sugar in Type 2 diabetes. In its latest results, presented at the American Diabetes Association conference, dulaglutide proved itself noninferior to Novo Nordisk's blockbuster Victoza--the $2-billion-a-year standard bearer among GLP-1 therapies. That's the first time a once-weekly GLP-1 agent has ever equaled a daily one in a Phase III trial, Lilly said--including GlaxoSmithKline's ($GSK) recently approved Tanzeum. Its easier treatment regimen will be at the heart of Lilly's campaign to sell this treatment as an alternative to Victoza. Lilly has a lot riding on the commercial success of this drug--failure is not an option. -- John Carroll
For more:
FDA hands Eli Lilly a big win, OKs dulaglutide for diabetes
Merck's weekly diabetes drug stacks up to blockbuster Januvia in Phase III
Lilly claims another head-to-head victory over Lantus in PhIII diabetes showdown
Lilly grabs a 'tentative' FDA OK for insulin treatment, but Sanofi still blocks its path
October
| 30. Harvoni | |
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Active ingredient: ledipasvir/sofosbuvir Disease: hepatitis C Peak sales estimate: more than $10 billion per year Approved: Oct. 10 Company: Gilead Sciences |
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The scoop: Gilead Sciences ($GILD) consistently outpaces its competition in developing next-generation hepatitis C therapies, and this year was no exception to the rule. The company in October won regulatory approval for Harvoni, a hep C combo drug that could cure the majority of patients without the need for painful injections. Gilead touts the product's promising numbers, as in three Phase III studies the drug boasted cure rates as high as 99.1% in patients with the genotype 1 variant of the virus. But Harvoni isn't the only all-oral hep C combo product making headway; Gilead faces competition from AbbVie's ($ABBV) three-drug cocktail with similar efficacy, and Merck's ($MRK) mix of MK-5172 and MK-8742 showed positive Phase II results. In the meantime, Gilead continues to ward off unhappy payers as coalition groups and PBMs decry the drug's $94,000 price tag for a 12-week course of treatment. -- Emily Wasserman
For more:
AbbVie vies for the hep C spotlight on the eve of its Gilead battle
Merck's hepatitis C cocktail drug fails a 4-week challenge
Payers hit back at Gilead for $94,500 price tag on brand-new hep C combo pill
Orphan drug pricing for non-orphan meds? Express Scripts is looking at you, Gilead
Gilead wins FDA approval for its hep C combo pill, a blockbuster in the making
Gilead nears EU approval for the next big thing in hep C
| 31. Akynzeo | |
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Active ingredients: netupitant and palonosetron Disease: chemotherapy-related nausea Approved: Oct. 10 Company: Eisai |
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The Scoop: With Akynzeo, Eisai and partner Helsinn took strides to expand its share of the market for treatments that mitigate the side effects of chemotherapy. The drug is a long-acting combination treatment designed to prevent nausea and vomiting among cancer patients undergoing chemo, blending palonosetron, approved in 2008 and sold by Eisai as Aloxi, with netupitant, a new agent. The former is designed to prevent nausea within the first 24 hours of chemo, while the latter provides relief for up to 5 days afterward. Aloxi, which brought in $428 million in the last fiscal year, competes with Merck's ($MRK) leading anti-emetic Emend, and Eisai figures its long-acting combo will be able to snag market share. -- Damian Garde
For more:
Eisai adds to chemo side-effects franchise with FDA nod for Akynzeo
| 32. Lumason | |
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Active ingredient: sulfur hexafluoride lipid microsphere Disease: ultrasound contrast agent for cardiac disease Approved: October 14 Company: Bracco Diagnostics |
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The Scoop: The original NDA for this contrast agent--used for patients whose echocardiograms are hard to read--dates all the way back to 2001 under the name SonoVue. Bracco, though, had run into safety issues raised by the FDA around ultrasound contrast agents and withdrew the NDA in 2007. But investigators were finally able to complete this 13-year odyssey with data from three studies involving 191 patients, with physicians attesting to the fact that they could use the technology to visualize the left ventricle and the inside of the chambers of the heart more clearly. Two other ultrasound contrast agents are already on the market. Approved in 39 countries ahead of the U.S., Bracco says that there are some 28 million echocardiograms each year in the U.S., with about 1 in 10 reported as "suboptimal." -- John Carroll
For more:
FDA gives nod to new agent for heart ultrasound imaging
| 33. Ofev | |
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Active ingredient: nintedanib Disease: idiopathic pulmonary fibrosis (IPF) Approved: Oct. 15 Company: Boehringer Ingelheim |
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The Scoop: Thanks to a regulatory setback for Roche ($RHHBY) competitor Esbriet, Boehringer Ingelheim was able to play catch-up and snag a green light for IPF drug Ofev on the same day as its rival to make the pair the first-ever approved treatments for the lung disease. Since then, the German pharma has rolled out a patient support program and copay assistance for the $96,000 orphan drug, which it hopes can help it build up some market share. Boehringer is relying on its marketing muscle, too, fielding a team of 100 to 200 sales reps and associated nursing professionals to get the word out as it gears up for the head-to-head showdown. -- Carly Helfand
For more:
Boehringer revs up Ofev sales force, support services to rival Roche's Esbriet in IPF
Same-day approvals send Roche, Boehringer to IPF market battle
With a new IPF drug on its way, Boehringer backs a Discovery documentary on the lung disease
| 34. Esbriet | |
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Active ingredient: pirfenidone Disease: idiopathic pulmonary fibrosis Peak sales estimate: $1.4 billion Approved: Oct. 15 Company: Roche |
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The Scoop: With Roche's ($RHHBY) $8.3 billion InterMune ($ITMN) buyout came Esbriet, a lung drug that U.S. regulators had already turned away once. Fast-forward less than two months and the Swiss pharma giant had itself an FDA green light--but so did its competitor. In a come-from-behind showing, Boehringer Ingelheim's Ofev won the agency's favor on the same day, setting up a market tug-of-war. But Roche has the "global resources and scale" to make for a swift Esbriet rollout, InterMune's CEO said in August, as well as a patient support program it hopes can help build up a market lead. It also has the head start in Europe, where Esbriet first launched in 2011. -- Carly Helfand
For more:
Boehringer revs up Ofev sales force, support services to rival Roche's Esbriet in IPF
Roche ready to roll on InterMune's lung drug with $8.6B buyout deal
InterMune takeover talk is tantalizing, but are investors too optimistic?
December
| 35. Blincyto | |
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Active ingredient: blinatumomab Disease: acute lymphoblastic leukemia Peak sales estimate: more than $500 million Approved: Dec. 3 Company: Amgen |
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The Scoop: Demonstrating its commitment to speedy reviews for promising cancer drugs, the FDA approved Amgen's ($AMGN) new drug more than 5 months ahead of schedule, putting the Big Biotech first in line among companies targeting a promising new oncology target. Blincyto is an antibody developed through Amgen's bispecific T cell engager (BiTE) system, designed to direct the body's disease-fighting immune cells to attack cancerous growth. The approval covers the relatively rare Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (ALL), and Amgen is running trials to widen its indication into other blood cancers. Blincyto's approval marks the first OK for any agent targeting CD19, a protein commonly found on cancer cells, leading a class that includes promising immunotherapies from Novartis ($NVS), Juno Therapeutics ($JUNO), Kite Pharma ($KITE) and others. And Amgen is hoping it's the first of many successes with BiTE technology, as the company is developing new candidates targeted against a wealth of malignancies. -- Damian Garde
For more:
Amgen snags a lightning-fast approval for its leukemia immunotherapy
Amgen pushes for early FDA OK of leukemia drug blinatumomab
Angling for early OK, Amgen wins 'breakthrough' title for leukemia drug
| 36. Xtoro | |
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Active ingredient: finafloxacin otic suspension Disease: swimmer's ear Approved: Dec. 17 Company: Alcon Labs |
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The Scoop: Technically, the condition that Xtoro targets is acute otitis externa, an infection of the outer ear and ear canal better known in the world as swimmer's ear. The therapy belongs to a class of fluoroquinolone antimicrobial drugs that can be used to treat infections caused by Pseudomonas aeruginosa and Staphylococcus aureus. There are already several on the market and the FDA was happy to make one more available for physicians to choose from. Fort Worth-based Alcon presented data from 560 patients, demonstrating that the treatment was able to resolve 70% of infections, compared to the 37% resolved by the solution used to deliver the drug. -- John Carroll
For more:
FDA approves new swimmer's ear drug
| 37. Viekira Pak | |
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Active ingredients: ombitasvir, paritaprevir and dasabuvir with the previously approved ritonavir. Disease: hepatitis C Peak sales estimate: $3 billion Approved: Dec. 19 Company: AbbVie |
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The Scoop: Rarely does a second approval in a new drug class get the kind of attention that AbbVie ($ABBV) earned for Viekira Pak. In many ways its cocktail drug leaves a lot to be desired when compared to Gilead's ($GILD) pioneering combo Harvoni. Among other things, it requires multiple doses every day and often requires ribavirin as an add-on. But with a cure rate in some populations reaching up to 99%, payers got the competing interferon-free remedy they were looking for. AbbVie offered only a modest wholesale discount on the drug, but stirred a tempest when the big pharmacy benefit manager Express Scripts announced that they had inked a deal to make Viekira Pak its sole hep C drug on its formulary in exchange for an unspecified "deep discount." You can be sure that other payers are anxious to negotiate the same kind of deal, which may leave Gilead's superior treatment far more marginalized far earlier than analysts had been anticipating. Merck ($MRK) has another hep C drug coming along in the clinic that will likely make this field even more competitive. -- John Carroll
For more:
AbbVie needs more than its hep C nod to backfill Humira's looming patent loss
AbbVie wins blockbuster hep C approval, starts wheeling and dealing on price
| 38. Lynparza | |
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Active ingredient: olaparib Disease: ovarian cancer Peak sales estimate: $2 billion (according to AstraZeneca) Approved: Dec. 19 Company: AstraZeneca |
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The Scoop: Last summer, when AstraZeneca ($AZN) made its pitch for an accelerated approval of olaparib, outside experts advised the FDA (11 to 2) to turn its back on the appeal. Olaparib had already failed a study for ovarian cancer--overall survival in the olaparib arm was 34.9 months compared to 31.9 months for placebo--and was later shelved, spurring a $285 million writeoff. But the new turnaround team that took charge under CEO Pascal Soriot wanted to take a second shot, pegging their argument on a retrospective analysis of the data showing a distinct progression-free survival benefit for a group of patients with germline BRCA mutations. After the panel vote, AstraZeneca came back with new data to show that the drug demonstrated an effect on a particular set of patients who had failed multiple therapies. The approval may make olaparib only a last-chance effort among a small set of desperately ill patients, but it's a start. -- John Carroll
For more:
AstraZeneca eyes $2B in sales with FDA nod for ovarian cancer fighter Lynparza
AstraZeneca grabs a surprise early approval for its $2B cancer contender
| 39. Zerbaxa | |
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Active ingredients: ceftolozane and tazobactam Disease: Gram-negative bacteria Peak sales estimate: $1 billion-plus Approved: December 22 Company: Cubist Pharmaceuticals |
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The Scoop: Cubist scored this approval just before its $9.5 billion M&A deal with Merck ($MRK) was set to go through. That's a particularly big plus for Merck, which some analysts suspect paid a bit too much for Cubist. But it's also the biotech's second approval this year, and it enjoys some added benefits lawmakers laid out to help encourage the development of new antibiotics. Federal incentives included a quick review for the application and an extra 5 years for patent exclusivity. Cubist's lead product is Cubicin, which is currently vulnerable to generic competition in mid-2016. Cubist recently started marketing Sivextro. -- John Carroll
For more:
Cubist has its Zerbaxa OK. Can Merck make the launch justify its $9.5B buyout?
Cubist scores another FDA nod on the eve of its $9.5B Merck merger
Looking for a bright side to the Merck-Cubist merger? It's the power of the sales rep
| 40. Rapivab | |
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Active ingredient: peramivir Disease: acute influenza Peak sales estimate: N/A Approved: December 22 Company: BioCryst |
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The Scoop: The FDA brought this therapy back from the dead last year. BioCryst had been ready to write it all off as a total loss, but then the FDA laid out a pathway to an approval and BARDA came up with the rest of the funds needed to gain an approval. With a pedigree like that, it's no wonder that analysts have only modest expectations for an order to fill the federal stockpile. -- John Carroll
For more:
Back from the scrap heap, BioCryst wins OK for IV flu drug
BioCryst's failed flu drug gets another shot at FDA success
BioCryst's shares spike on hope that flu drug can rise from the grave
| 41. Opdivo | |
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Active ingredient: nivolumab Disease: melanoma Peak sales estimate: $5 billion to $8 billion Approved: December 22 Company: Bristol-Myers Squibb |
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The Scoop: The FDA had to move up its decision day by three months to squeeze this approval into a record-breaking year. Opdivo comes just one step behind Merck's ($MRK) Keytruda, leaving the two to battle it out to win the first OK for lung cancer, where Bristol-Myers Squibb ($BMY) is seen as the leader to beat. Both drugs block PD-1, qualifying them as the pioneering checkpoint inhibitors on the market. PD-1 and the related target PD-L1 have become one of the biggest focuses in immuno-oncology today. Not only do they work on their own, they're natural additions to a new wave of combinations now in the clinic. And they're tied to significant gains in efficacy along with a relatively clean safety profile. Coming up behind Bristol-Myers are Roche and AstraZeneca ($AZN), and then a long lineup of biopharma players looking to play catch-up. In many ways melanoma is simply the beachhead for this field. There's lots more sales territory yet to conquer. -- John Carroll
For more:
With Opdivo approval, Bristol-Myers arms for PD-1 battle against Merck's Keytruda
Bristol-Myers' pioneering PD-1 drug Opdivo OK'd by FDA for melanoma
Merck's PD-1 star Keytruda shines at ASH, but lags behind Bristol-Myers' rival