After disappointing 2016, new drug approvals roared back to life in 2017

fda

New drug approvals all but screeched to a halt in 2016, with a measly 22 new OKs after a pair of bountiful years. But the FDA wasted no time in 2017, signing off on 12 drugs in the first quarter alone—besting its record for the same period of any year in recent history.

All told, the agency cleared 46 new molecular entities*—surpassing its 2016 total in June—as well as a handful of key biologics, a new shingles vaccine and a pair of CAR-T therapies for blood cancers.

While the FDA itself has pooh-poohed any suggestions that it has grown more lenient, industry watchers aren’t buying it, EP Vantage wrote in its 2018 Preview. In fact, the agency’s “U-turns” on rigorous safety requirements in drugs, such as Eli Lilly’s rheumatoid arthritis drug Olumiant, and its controversial decision to approve Sarepta’s Duchenne drug Exondys 51 last year are helping drive the perception of a “friendly FDA.”

Novartis and AstraZeneca emerged the clear winners, with three approved therapies each. Pfizer, Roche, Valeant and tandem Sanofi and Regeneron each scored two approvals. Gilead also logged two, if you count Yescarta, picked up in its Kite Pharma buyout, and GlaxoSmithKline grabbed one in the form of shingles vaccine Shingrix. Bristol-Myers was the only top 15 pharma company to go without in 2017.

While cancer drugs notched 14 approvals, most of these targeted specific subtypes of cancer. Novartis’ Kisqali and Eli Lilly’s Verzenio, for example, were approved for patients with HR-positive, HER2-negative breast cancer, and Celgene and Agios’ Idhifa is for treating relapsed or refractory acute myeloid leukemia in patients with a particular mutation.

Pfizer and Merck KGaA’s cancer drug Bavencio beat AstraZeneca’s Imfinzi to approval, but its first indication, the rare skin cancer Merkel cell carcinoma, won’t make a dent in its $4 billion peak sales estimate. The partners are hoping to make it past the FDA in a number of other indications and are testing the drug in kidney cancer, ovarian cancer, non-small cell lung cancer and others.

A Duchenne drug took the crown for most controversial nod for the second year running. Marathon Pharmaceuticals came under fire for slapping an $89,000-per-year price tag on the corticosteroid Emflaza, which had already been available overseas for decades for as little as $1,000 annually. The company then looked for a quick payout by offloading the drug to PTC Therapeutics, which is aiming to lower its price to around $35,000 a year.

2017 saw some firsts, including the first FDA-approved drugs for the movement disorder tardive dyskinesia: Neurocrine’s Ingrezza and Teva’s Austedo. And Tesaro’s Zejula sped through FDA review, becoming the only PARP inhibitor that treats all women with recurrent ovarian, fallopian tube or peritoneal cancer, not just women with a BRCA mutation.

Other diseases gained their first new drug in a while: Newron’s Xadago was the first FDA-approved Parkinson’s drug in more than a decade, and Mitsubishi Tanabe’s Radicava is the first new ALS treatment in 22 years.

Novartis’ Kymriah and Kite’s—now Gilead’s—Yescarta made history as the first CAR-T therapies to win approval. Cleared for two different blood cancers, the treatments require a patient’s T cells to be harvested, engineered to fight cancer and then reinfused back into the patient, costing $475,000 and $373,000, respectively. Industry watchers will be paying close attention to their uptake to “evaluate the future value of these and rival therapies,” and to gauge whether Gilead’s $12 billion bet on Kite was worth it, EP Vantage said.

“Huge leaps forward in cancer care with the anti-PD-(L)1 antibodies and the approval of cutting-edge techniques like CAR-T have raised expectations that the sector can keep delivering at pace,” said EP Vantage.

But it cautioned that “excitement” over the long-term gains in up-and-coming fields—such as gene therapy and RNA-based treatments—should be “tempered by realism about the short-term challenges.” Nonetheless, the outlook is positive.

“If you look at the pace of R&D, there has probably never been a better time for a lot of science,” Ben Yeoh, senior portfolio manager at RBC Global Asset Management, said in the EP Vantage report. “We’re curing cancers that we’ve never cured before, we’ve got new drugs for MS, new treatments for diabetes. Costs aside, we are still making progress on human health.”

Without further ado, here's our list of drugs that earned FDA approval in 2017.

* FierceBiotech has historically included only new molecular entities in this report. This year, we have included a few more, including a vaccine, biologics and the CAR-T therapies.

1. Trulance

2. Parsabiv

3. Emflaza

4. Siliq

5. Xermelo

6. Odactra

7. Kisqali

8. Xadago

9. Bavencio

10. Symproic

11. Zejula

12. Dupixent

13. Ocrevus

14. Austedo

15. Ingrezza

16. Brineura

17. Alunbrig

18. Rydapt

19. Tymlos

20. Imfinzi

21. Radicava

22. Kevzara

23. Rebinyn

24. Fibryna

25. Baxdela

26. Haegarda

27. Bevyxxa

28. Tremfya

29. Nerlynx

30. Vosevi

31. Idhifa

32. Mavyret

33. Besponsa

34. KedRAB

35. Vabomere

36. Benznidazole

37. Kymriah

38. Solosec

39. Aliqopa

40. Verzenio

41. Yescarta

42. Shingrix

43. Calquence

44. Vyzulta

45. Prevymis

46. Fasenra

47. Mepsevii

48. Hemlibra

49. Ozempic

50. Xepi

51. Rhopressa

52. Luxturna

53. Steglatro

54. Macrilen

55. Giapreza

1. Trulance

Active ingredient: plecanatide
Disease: chronic idiopathic constipation
Peak sales estimate: $500 million to $1 billion
Approved: Jan. 19
Company: Synergy Pharmaceuticals

The scoop: Synergy’s Trulance is going after Takeda-Sucampo’s Amitiza and Allergan-Ironwood’s market leader Linzess in chronic idiopathic constipation. One edge Trulance has is that it can be taken with or without food, potentially boosting adherence. Soon after—and even before—it became the first drug approved by the FDA in 2017, Synergy made a strenuous effort to secure it a successful trajectory. In 2015, the company tapped Shire and Johnson & Johnson veteran Troy Hamilton, who has extensive commercial experience in gastroenterology, as chief commercial officer, and in 2017 adopted a hybrid sales organization of about 250 people that includes both in-house sales managers and contract reps. It priced Trulance on par with competitors, rolled out a novel disease campaign featuring a “Poop Troop” of emojis, and used a stock offering and debt financing to support Trulance-related activities. It also secured three patents that would protect Trulance until 2032. But those efforts hit a snag, as Express Scripts excluded the medication from its national preferred formulary for 2018. Synergy is looking to expand the battle with Linzess into IBS-C, having submitted an sNDA for Trulance in the indication with the PDUFA date coming soon in January. — Angus Liu

2. Parsabiv

Active ingredient: etelcalcetide
Disease: secondary hyperparathyroidism
Peak sales estimate: N/A
Approved: Feb. 8
Company: Amgen

The scoop: On its first go-around, the FDA rejected Amgen’s Parsabiv, a treatment for secondary hyperparathyroidism in adult patients with chronic kidney disease on hemodialysis. In 2016, the FDA issued a complete response letter for the drug for reasons that Amgen never disclosed. Whatever the reason, the Thousand Oaks, California-based biotech was able to quickly resolve the matter and won an approval six months later. Amgen picked the drug up with its $315 million deal in 2012 for KAI Pharmaceuticals. Parsabiv works as a calcimimetic agent, a drug designed to mimic the action of calcium by activating the calcium-sensing receptors on the parathyroid gland. Parsabiv, which is given intravenously, binds to and activates the calcium-sensing receptor on the parathyroid gland, decreasing levels of parathyroid hormone. In three phase 3 trials posted in 2015, the drug met its primary goals by significantly reducing parathyroid hormone levels. Amgen claims it has the advantage over other treatments because it is the only calcimimetic that can be administered intravenously by the dialysis healthcare team three times a week at the end of the hemodialysis session. — Eric Palmer

3. Emflaza

Active ingredient: deflazacort
Disease: Duchenne muscular dystrophy
Peak sales estimate: $100 million
Approved: Feb. 9
Company: Marathon Pharmaceuticals

The scoop: Likely the most controversial approval of the year, Marathon Pharmaceuticals’ corticosteroid Emflaza inflamed pressures on the industry and raised questions about whether the "new FDA" under President Donald Trump was being a little too lenient. Why? Well, because the drug is just an old steroid that hadn’t been approved in the U.S., but Marathon managed to get it in for Duchenne muscular dystrophy (DMD), a rare condition that causes muscle wasting in young boys, and then slapped an $89,000 price tag on it. In the U.K., where it has been available for years, it costs just under £16, or $21, for a pack of 60 6-mg tabs. On top of this, it also won seven years of orphan drug exclusivity. Then, a month later, it sold the drug to PTC Therapeutics for as much as $190 million; the biotech, which has struggled to bring its own DMD medication to the U.S. market, lowered the price to $35,000, but the whole saga left many with a bitter taste. — Ben Adams

4. Siliq

Active ingredient: brodalumab
Disease: psoriasis
Peak sales estimate: N/A
Approved: Feb. 15
Company: Valeant

The scoop: After brodalumab developers Amgen and AstraZeneca turned up suicides in clinical trials, Amgen walked away from the candidate. A few months later, AstraZeneca was out, too, having shipped rights on the prospect to Valeant for $100 million upfront. Valeant, though, plowed ahead, eventually snagging an FDA approval. But the go-ahead came along with a black-box warning, the agency’s most serious, as well as a risk-management program that Evercore ISI analyst Umer Raffat described at the time as “onerous.” And unfortunately for Valeant, those are burdens that competitors Cosentyx from Novartis, Taltz from Eli Lilly and Tremfya from Johnson & Johnson—which came along more recently—aren’t saddled with. — Carly Helfand

5. Xermelo

Active ingredient: telotristat ethyl
Disease: carcinoid syndrome diarrhea
Peak sales estimate: $350 million
Approved: Feb. 28
Company: Lexicon Pharmaceuticals

The scoop: Lexicon launched Xermelo, the first drug approved in the company’s 22-year history, right after it was approved. It is used in combination with the standard-of-care somatostatin analogue (SSA) therapy—including Ipsen’s Somatuline and Novartis’ Sandostatin—for the rare condition that happens in some people with metastatic neuroendocrine tumors. The drug was off to a good start. Within a month, 19 of the top 20 high-volume centers that prescribe SSA therapy had written a Xermelo script, according to company CCO Alex Santini during the first-quarter earnings call. By the end of June, 443 patients were on Xermelo, and that number increased to 685 through Sept. 30. “Of the prescribing physicians, the majority indicate that Xermelo is meeting or exceeding their expectations,” Santini said during the third-quarter call. With an annual price tag of $61,000 to $72,000, the drug is expected to reach $350 million in peak U.S. sales. Under an ex-U.S. licensing deal, Ipsen obtained European approval in September. — Angus Liu

6. Odactra

Active ingredient: allergen extract
Disease: house dust mite allergy
Peak sales estimate: N/A
Approved: March 1
Companies: Merck & Co., ALK-Abello

The scoop: Odactra is the first sublingual treatment for allergic reactions to dust mites, providing an alternative to allergy shots to fight off the nasal congestion and watery eyes the microscopic bugs induce in some people. The license was granted to Merck & Co. but transferred a few months later to Denmark-based ALK. That is because of Merck’s surprise decision last year to return the rights to three sublingual allergy immunotherapy tablets it developed with ALK, including Odactra. The other two were for tablets for fighting grass and ragweed allergies. Merck had paid out more than $100 million to ALK as part of the deal it struck in 2007 for the rights to the drugs, but faced with healthcare professionals who were reluctant to shift away from existing treatment options, Merck struggled to gain ground. In August, the FDA transferred the license to ALK, which said it expected to launch the pills in the U.S. yet this year. — Eric Palmer

Kisqali

7. Kisqali

Active ingredient: ribociclib
Disease: HR-positive, HER2-negative breast cancer
Peak sales estimate: $1.6 billion
Approved: March 13
Company: Novartis

The scoop: In March, Novartis’ Kisqali became the first-in-class rival to Pfizer’s Ibrance, a blockbuster with a two-year market lead. And it did so with another disadvantage, too: a recommendation for ECG monitoring, thanks to mild cardiac arrhythmias and one drug-related sudden death that turned up in clinical trials. Novartis, which undercut Ibrance on price with a flexible structure tied to dose side, has insisted the monitoring is “relatively routine.” But that doesn’t mean it’s had much success chipping away at Pfizer’s lead. Through three quarters, the product had captured just 5% of total prescriptions, CEO Joe Jimenez told investors on October’s third-quarter conference call. — Carly Helfand

8. Xadago

Active ingredient: safinamide
Disease: Parkinson's disease
Peak sales estimate: $500 million
Approved: March 21
Company: Newron Pharmaceuticals

The scoop: Xadago had a long and troubled journey to approval as a complementary treatment for patients taking levodopa/carbidopa, specifically to be used during “off” episodes, or when these primary drugs don’t work as well. Xadago faced a phase 3 failure back in 2010 and subsequently, Newron’s relationship with then-partner Merck KGaA fell through and the company lost a $63 million buyout offer from Finland’s Biotie. Newron submitted the drug for approval in 2014, but the FDA rejected the bid in March last year, leading the company to resubmit in September. Turns out the second time was the charm: In March, Xadago bagged the FDA nod, the first new Parkinson’s drug to do so in more than a decade. — Amirah Al Idrus

Bavencio

9. Bavencio

Active ingredient: avelumab
Disease: cancer
Peak sales estimate: $4 billion
Approved: March 23
Companies: Pfizer, Merck KGaA

The scoop: As a latecomer into the immuno-oncology checkpoint inhibitor market, Merck KGaA and partner Pfizer’s Bavencio (avelumab) is still hoping to make billions at peak, despite competition from Merck’s Keytruda, Bristol-Myers Squibb’s Opdivo and Roche’s Tecentriq, as well as most recently AstraZeneca’s Imfinzi. All of its competition has, however, been beset by postmarketing setbacks over the last year as they undertook various attempts to broaden the reach of their drugs into different settings and cancers. Bavencio’s first approval, in the rare skin cancer Merkel cell carcinoma, will not help it reach the $4 billion peak sales estimate, but the pair are studying Bavencio in non-small cell lung cancer, kidney cancer, ovarian cancer, and others, and it’s testing the drug alongside some of Pfizer’s in combination approaches. In May, it also gained a speedy FDA approval in urothelial carcinoma. — Ben Adams

10. Symproic

Active ingredient: naldemedine
Disease: opioid-induced constipation
Peak sales estimate: N/A
Approved: March 23
Companies: Shionogi, Purdue Pharma

The scoop: A latecomer to the opioid-induced constipation market, behind AstraZeneca’s Movantik and Valeant’s Relistor, Japan’s Shionogi and Connecticut’s Purdue Pharma have an uphill sales battle for their oral, peripherally acting mu opioid receptor antagonist Symproic. First, it’s coming from behind in the market and has also been hit with an addiction risk red flag from the Drug Enforcement Administration as a Schedule II controlled substance. It will hope to have that removed, but sales for its rivals have been sluggish, with Movantik and Relistor bringing home revenue in the tens of millions. — Ben Adams

niraparib

11. Zejula

Active ingredient: niraparib
Disease: Recurrent ovarian, fallopian tube or peritoneal cancer
Peak sales estimate: $1.9 billion
Approved: March 27
Company: Tesaro

The scoop: Zejula won FDA approval months before its expected action date at the agency, giving its maker Tesaro a pleasant surprise back in March. With strong backing data, the PARP inhibitor also won a broad label, bolstering its chances to shake up a market previously occupied by AstraZeneca's Lynparza and Clovis Oncology's Rubraca. And that's just what it did. Just a few months after launch, the drug rocketed to the top position in the PARP inhibitor field, holding 60% of share. The FDA approved Zejula to treat all women with recurrent ovarian, fallopian tube or peritoneal cancer who’ve previously responded to platinum chemo, not just those who test positive for the BRCA genetic mutation. Analysts have predicted $1.9 billion in sales by 2022, making it among 2017's top launches. — Eric Sagonowsky

Dupixent package

12. Dupixent

Active ingredient: dupilumab
Disease: atopic dermatitis
Peak sales estimate: $3 billion
Approved: March 28
Companies: Sanofi, Regeneron

The scoop: Sanofi's biggest drug approval in recent years came this spring when the FDA signed off on Dupixent, an IL-4 and IL-13 inhibitor and the first atopic dermatitis treatment to reach the market in several years. Carrying a $3 billion peak sales projection in that disease and follow-on indications such as asthma and severe nasal allergies, Dupixent launched with a $37,000 price tag and has been hitting its stride in the market. Ahead of the launch, Sanofi preemptively sued Amgen, asking a court to recognize that its blockbuster-to-be didn't infringe on an Amgen patent. In the first nine months of 2017, Dupixent generated €101 million ($117 million) in sales. Behind Roche's multiple sclerosis entrant Ocrevus, Dupixent is the second biggest drug launch of 2017, according to Evaluate data on sales through 2022. — Eric Sagonowsky

13. Ocrevus

Active ingredient: ocrelizumab
Disease: multiple sclerosis
Peak sales estimate: $4.1 billion
Approved: March 28
Company: Roche

The scoop: After a three-month review delay to work through details about manufacturing, Roche's multiple sclerosis game-changer won FDA approval in March. The drug garnered approval to treat relapsing-remitting MS and the harder-to-treat primary progressive form in adult patients, and Switzerland's Roche surprised the market by pricing its new therapy at a discount. Roche tagged Ocrevus with a $65,000-per-year list price, a 25% discount to Merck KGaA's Rebif and a 20% discount to other drugs, according to an analyst. A spokesperson said the company made the decision in order to "reverse the trend" of skyrocketing MS drug prices, which have grown several times over in recent years. In response, a Merck KGaA spokesperson dismissed the comparison as "misleading and oversimplified." The discount seems to have worked for the launch: The drug generated $200 million in sales during its first quarter on the market. With a $4.1 billion sales projection by 2022, it's expected to be pharma's biggest launch of 2017. — Eric Sagonowsky

Austedo

14. Austedo

Active ingredient: deutetrabenazine
Disease: chorea associated with Huntington’s
Peak sales estimate: $700 million
Approved: April 3
Company: Teva

The scoop: Teva’s Austedo was just the second ever to win an approval to treat chorea, the involuntary, random and sudden movements that occur in about 90% of Huntington’s disease patients. And it was also the cheapest. Teva rolled out the product at a list price of $60,000 per year, less than half of the wholesale cost of competitor Xenazine from Valeant and far below even the prices for tetrabenazine generics, which run between $92,000 and $96,000. In August, the Israeli drugmaker followed up with Austedo’s second FDA nod, this one for tardive dyskinesia, a movement disorder that had no agency-approved treatments until Neurocrine’s Ingrezza showed up in April. — Carly Helfand

15. Ingrezza

Active ingredient: valbenazine
Disease: tardive dyskinesia
Peak sales estimate: $1.3 billion
Approved: April 11
Company: Neurocrine

The scoop: When Neurocrine grabbed its FDA approval for Ingrezza, the drug became the first ever to win an indication in involuntary movement disorder tardive dyskinesia. But Ingrezza’s solo status on the market didn’t last. In August, Teva’s Austedo picked up its own nod to give Ingrezza a little competition. Neurocrine, though, is aiming for another market, too, and that’s Tourette syndrome. But its ambitions in that space took a hit in March when Ingrezza flopped a phase 2 study in pediatric patients, failing to outdo placebo in registering a change on the Yale Global Tic Severity Scale. The news wasn’t all bad, though. Company execs said they’d underestimated the dose needed to deliver benefits in kids, and that they’d kick-start a new-and-improved study with the findings in mind. “The program is not dead, in our view,” Leerink Partners analyst Paul Matteis wrote to clients at the time. — Carly Helfand

16. Brineura

Active ingredient: cerlipnase alfa
Disease: CLN2 disease
Peak sales estimate: $100 million to $200 million
Approved: April 27
Company: BioMarin Pharmaceutical

The scoop: With a list price before discounts of $702,000 per year, BioMarin’s rare disease drug Brineura ranks among the world’s most expensive. After discounts, the company has said that on federal programs, it will cost just $486,000 per patient per year, and they say most of the candidates for the drug are on federal programs. The drug is the first to treat the disease, and BioMarin execs have said the price is justified by the innovation factor and the “huge unmet need.” The medication is approved to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). In the U.S., the disease affects fewer than one in 1 million children, about 22 a year, but CLN2 is a nasty condition. It is a rapidly progressive, fatal brain condition in which children can be expected to completely lose the ability to walk and talk at around 6 years of age and die between the ages of 8 and 12. The approval requires BioMarin to conduct follow-up safety studies in patients under 2 years of age, plus a long-term study tracking real-world safety over a minimum of 10 years. BioMarin has told investors that given the rareness of the disease, and that many children with the condition have not yet been diagnosed, it will take time to build sales. Perhaps more valuable in the short term is the priority review voucher that BioMarin won with the Brineura approval, a significant award that could be worth hundreds of millions of dollars in resale to another biopharma. — Eric Palmer

17. Alunbrig

Active ingredient: brigatinib
Disease: ALK-positive, non-small cell lung cancer
Peak sales estimate: $1 billion-plus
Approved: April 28
Companies: Ariad, Takeda

The scoop: Biopharma-backed Takeda spent $5.2 billion on its Ariad buy at the start of the year and got an immediate payoff when the biotech gained a second-line lung cancer approval in patients with the ALK mutation from the FDA a few short months later. CEO Christophe Weber said his company believes Alunbrig “will become a best-in-class ALK inhibitor … with the potential to achieve peak annual sales of over $1 billion.” These patients are in line for the treatment if they fail on Pfizer and Merck KGaA’s ALK drug Xalkori, although here it will need to compete with Roche’s Alecensa and Novartis’ Zykadia. But, as is often the case with first approvals in a lower setting, Takeda is hoping to bump up its drug to first-line in late-stage tests by taking on Xalkori directly and potentially boost its blockbuster potential. — Ben Adams

Rydapt

18. Rydapt

Active ingredient: midostaurin
Disease: acute myeloid leukemia
Peak sales estimate: $260 million by 2019
Approved: April 28
Company: Novartis

The scoop: The FDA approved midostaurin for patients that have acute myeloid leukemia (AML) and the specific genetic mutation called FLT3. The kinase inhibitor was approved for use with a companion diagnostic and in combination with chemotherapy. The drug was also approved for adults with certain types of rare blood disorders. AML accounts for about 25% of all adult leukemias, according to Novartis, and has the lowest survival rate. Rydapt charted a median overall survival of 74.7 months in a phase 3 trial, beating out the 25.6 months seen on placebo alone. — Eric Palmer

19. Tymlos

Active ingredient: abaloparatide
Disease: osteoporosis in postmenopausal women
Peak sales estimate: $450 million
Approved: April 28
Company: Radius Health

The scoop: When Radius first won approval for Tymlos, at least one analyst thought the medication wouldn’t be able to stand up to Eli Lilly’s incumbent Forteo and Amgen’s forthcoming romosozumab. But since then, things have turned dramatically in Radius’ favor. For one, the FDA rejected Amgen’s candidate after it turned up negative cardiovascular data in May. And for two, PBM giant Express Scripts booted Forteo from its 2018 national preferred formulary, listing Tymlos among its preferred alternatives. Now, it’s up to new CEO Jesper Høiland, former president of Novo Nordisk’s U.S. business, to navigate the launch. He’s got 230 reps and 22 medical science liaisons signed on for the job. — Carly Helfand

20. Imfinzi

Active ingredient: durvalumab
Disease: urothelial carcinoma
Peak sales estimate: $2.8 billion
Approved: May 1
Company: AstraZeneca

The scoop: AstraZeneca's Imfinzi earned its way onto the market back in May with an initial nod in advanced bladder cancer, following Roche's Tecentriq and Bristol-Myers Squibb's Opdivo into the field. The approval represented a foray for AstraZeneca into the lucrative checkpoint inhibitor field, already dominated by Opdivo and Merck's Keytruda. Shortly after Imfinzi's FDA nod in bladder cancer, Keytruda won a nod in the disease area, as well as Pfizer and Merck KGaA's Bavencio. Aside from bladder cancer, AstraZeneca is testing its checkpoint inhibitor in a host of other cancer types across various trial stages. It's also awaiting an FDA decision in the important treatment area of lung cancer maintenance and is testing the drug in a combo with CTLA-4 antibody candidate tremelimumab in first-line lung cancer. Bernstein analyst Tim Anderson, M.D., estimates $2.8 billion in sales by 2022. — Eric Sagonowsky

Radicava

21. Radicava

Active ingredient: edaravone
Disease: amyotrophic lateral sclerosis
Peak sales estimate: N/A
Approved: May 5
Company: Mitsubishi Tanabe

The scoop: Radicava posted such compelling data in Japanese trials that the FDA invited Mitsubishi Tanabe to file for U.S. approval on the same data. The drug earned approval via the FDA’s orphan drug review program, the first new amyotrophic lateral sclerosis (ALS) drug in 22 years. And while it doesn’t halt or reverse ALS—no available drug does—it slows the disease’s progression by 33%, according to phase 3 data. Radicava beat two promising candidates—AB Science’s masitinib and Cytokinetics' tirasemtiv—to FDA approval. The treatment, administered intravenously every day for two weeks, followed by two weeks without the drug, costs $1,086 a pop. Subsequent infusion cycles last 10 to 14 days and are followed by the requisite two drug-free weeks, bringing the treatment’s cost to $145,524 per year before discounts. — Amirah Al Idrus

22. Kevzara

Active ingredient: sarilumab
Disease: rheumatoid arthritis
Peak sales estimate: $1 billion
Approved: May 22
Companies: Sanofi, Regeneron

The scoop: Sanofi and Regeneron's productive R&D partnership yielded yet another new drug in May with the FDA approval for rheumatoid arthritis treatment Kevzara. The partners' second immunology entry for the year behind Dupixent, Kevzara carries a $39,000 list price and is set to do battle with AbbVie's Humira, the world's bestselling drug, plus Roche's older Actemra. But the path to market wasn't without problems. The FDA rejected the drug last October due to manufacturing issues, sending the companies scrambling to fix them. Nonetheless, they passed an inspection early this year, leading the market to believe an approval was imminent. The drug is carrying blockbuster expectations at a time when Sanofi's diabetes business has been taking a beating, leading the company to lean heavily on new therapies for growth. Beyond rheumatoid arthritis, Sanofi is hoping to expand the drug into other uses, such as uveitis, for which it's currently in a phase 2 study. — Eric Sagonowsky

23. Rebinyn

Active ingredient: nonacog beta pegol, N9-GP
Disease: hemophilia B
Peak sales estimate: N/A
Approved: May 31
Company: Novo Nordisk

The scoop: While Novo’s name is synonymous with insulin products, the Danish drugmaker also has a small portfolio of hemophilia products that generate about $1.7 billion a year. It is into this group that Rebinyn fits. About 5,000 people a year are diagnosed in the U.S. with hemophilia B, in which a lack of blood clotting factor IX can lead to bleeding episodes. Rebinyn is approved for on-demand treatment and control of bleeding episodes, as well as to manage bleeding before, during or after surgery in adults and children with hemophilia B. Its approval should help offset some generic sales erosion for one of Novo's older hemophilia drugs, NovoSeven. — Eric Palmer

24. Fibryna

Active ingredient: fibrinogen
Disease: congenital fibrinogen deficiency
Peak sales estimate: N/A
Approved: June 7
Company: Octapharma Pharmazeutika Produktionsgesellschaft

The scoop: Lachen, Switzerland-based Octapharma, which already makes other treatments for bleeding disorders, won approval for its human fibrinogen replacement to treat acute bleeding episodes in adults and adolescents who are at least 12 years of age and who suffer from congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. The condition is a very rare, inherited blood disorder in which the blood does not clot normally. Fibryna will be manufactured at the company's site in Vienna, Austria. — Eric Palmer

25. Baxdela

Active ingredient: delafloxacin
Disease: acute bacterial skin and skin structure infections
Peak sales estimate: $400 million in ABSSSI
Approved: June 19
Company: Melinta Therapeutics

The scoop: After spending many years and tens of millions of dollars in the R&D stage, Melinta Therapeutics won its first drug approval this year in Baxdela to treat acute bacterial skin and skin structure infections. The antibiotic, delafloxacin, is available in either oral or IV formulations and fights both gram-positive and gram-negative pathogens, according to the company's approval announcement. Because the drug is a Qualified Infectious Disease Product, Melinta gets an extra five years of exclusivity for its antibiotic; its patents expire between 2025 and 2029, according to the FDA Orange Book. Cempra, which merged with Melinta in August, said in an investor presentation that Baxdela could bring in $400 million in the approved indication alone. Further, the combined company is also evaluating Baxdela in a phase 3 trial in community-acquired bacterial pneumonia and plans a new trial in patients with complicated urinary tract infections. In 2014, Melinta abandoned a plan to explore a use in gonorrhea, instead switching to ABSSSI. — Eric Sagonowsky

26. Haegarda

Active ingredient: C1 esterase inhibitor
Disease: hereditary angioedema
Peak sales estimate: N/A
Approved: June 22
Company: CSL Behring

The scoop: CSL Behring’s Haegarda is the first treatment for hereditary angioedema (HAE) attacks that can be administered subcutaneously, making it easier for patients or caregivers to administer at home. But its approval wasn’t met entirely with applause. It also drew a lawsuit from Shire, which markets HAE medications Cinryze, Firazyr and Kalbitor, and it's working on a subcutaneous prophylactic C1 esterase inhibitor. The lawsuit, which claimed CSL violated a recently won patent, was trying to prevent CSL from launching Haegarda. So, how did the Australian company respond? It launched Haegarda at an 18% discount to Shire’s Cinryze, before discounts, clearly aiming to take a bite out of Cinryze’s market share. According to the FDA, between 6,000 and 10,000 people in the U.S. suffer from HAE, a condition caused by shortages of a plasma protein dubbed C1 esterase inhibitor. Patients can suffer from rapid swelling attacks that can happen spontaneously or be caused by stress, surgery or infection. — Eric Palmer

27. Bevyxxa

Active ingredient: betrixaban
Disease: prevention of venous thromboembolism
Peak sales estimate: $1.7 billion
Approved: June 23
Company: Portola Pharmaceuticals

The scoop: Even after it appeared to have faltered in a phase 3 trial, Bevyxxa won over U.S. regulators to become the first oral factor Xa inhibitor anticoagulant approved for preventing venous thromboembolism (VTE) in acute medically ill patients. Its secret? Developer Portola’s ability to reanalyze data from the study. Investigators focused on a group of D-dimer-positive patients and did exploratory analyses that showed a consistent and significant reduction in VTE with Bevyxxa across all study cohorts. With that, Credit Suisse analyst Vamil Divan, M.D., estimated an eventual $1.7 billion in sales. The drug could see competition from Johnson & Johnson’s well-established blood thinner Xarelto, which is also being tested for the indication. But there’s a more imminent threat. A draft guideline by clinicians and scientists against an entire group of anticoagulants for extended prophylaxis could complicate Bevyxxa’s launch. In November, the FDA extended the review period for a Prior Approval Supplement for Bevyxxa with a new action date of Jan. 30, 2018, but the company said it still expected a launch by February. — Angus Liu

Tremfya

28. Tremfya

Active ingredient: guselkumab
Disease: plaque psoriasis
Peak sales estimate: $1.5 billion
Approved: July 13
Company: Johnson & Johnson 

The scoop: Tremfya joins Novartis' Cosentyx, Eli Lilly’s Taltz and Valeant’s Siliq in a market that’s recently exploded with new-age options. The mAb works by targeting interleukin (IL)-23, a protein which has been shown to play a key role in the development of immune-mediated inflammatory disease, and despite the competition, is still expected by analysts at EvaluatePharma to be worth around $1.5 billion at peak. Cosentyx is still seen as the big boy in this market, however, and last January the Swiss major added two new approvals for their drug, for psoriatic arthritis and ankylosing spondylitis, as it guns for $5 billion in peak revenue. But Tremfya works differently from its peers: Where it goes for IL-23, its rivals target interleukin-17, and J&J is pitting Tremfya against Cosentyx in a head-to-head trial it hopes will prove the new drug superior. — Ben Adams

29. Nerlynx

Active ingredient: neratinib
Disease: HER2-positive breast cancer
Peak sales estimate: $1.25 billion+
Approved: July 17
Company: Puma Biotechnology

The scoop: Kinase inhibitor Nerlynx was approved to prevent recurrence of HER2-positive breast cancer in women previously treated with Roche’s Herceptin. While some FDA reviewers were skeptical about efficacy—and worried about side effects—analysts have predicted strong demand for the new drug. Puma never said publicly what the list price would be, but analysts have tagged it at around $10,600, a premium price that reflects the fact that the label was not restricted to hormone-receptor positive patients, even though the biggest benefit during clinical trials was seen in those women. Initially, Puma said it would launch the drug in September, raising eyebrows among some investors who thought a buyout of the Los Angeles-based biotech might be brewing, but when Puma advanced the launch to August, analysts cooled on that idea. — Eric Palmer

30. Vosevi

Active ingredients: sofosbuvir, velpatasvir and voxilaprevir
Disease: hepatitis C
Peak sales estimate: $1.1 billion
Approved: July 18
Company: Gilead Sciences

The scoop: Gilead’s hepatitis C franchise was already on the decline when Vosevi was approved for second-line treatment. Its $24,920-per-bottle list price is the same as Epclusa’s, but it contains a new compound on top of the two ingredients in Epclusa. As many first-line treatments have high cure rates, only a few patients will be eligible for Vosevi. But some industry watchers still think it has potential to reach blockbuster status. The problem, as Leerink Partners analyst Geoffrey Porges saw it, is that the U.S. approval doesn’t include an option in the EU approval that shortens therapy to eight weeks in some genotype 3 patients. Gilead’s hep C decline will only get worse now that AbbVie’s significantly cheaper Mavyret has been approved as an eight-week treatment. The overlap between Vosevi and Mavyret lies in Mavyret’s additional approval for HCV genotype 1 patients who have previously been treated with a regimen containing either an NS5A inhibitor (Daklinza) or an NS3/4A protease inhibitor (Olysio). — Angus Liu

Idhifa

31. Idhifa

Active ingredient: enasidenib
Disease: relapsed or refractory acute myeloid leukemia
Peak sales estimate: $500 million
Approved: Aug. 1
Companies: Celgene, Agios

The scoop: In 2010, Celgene inked a deal with Agios to assume global development and commercialization rights to a drug candidate that would eventually become the first approved drug for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with an IDH2 mutation. Idhifa snagged priority review in March and five months later, well before its PDUFA date of Aug. 30, scored the FDA nod. It is the first approved drug for the 8% to 19% of AML patients with this particular mutation, but it doesn’t come cheap. Its prediscount price is $24,872 per month, according to SunTrust analyst Yatin Suneja, exceeding the $14,000 number he had modeled. — Amirah Al Idrus

32. Mavyret

Active ingredients: glecaprevir and pibrentasvir
Disease: hepatitis C
Peak sales estimate: $1.25 billion
Approved: Aug. 3
Company: AbbVie

The scoop: In recent years, a burst of competition in the hep C space has killed pricing power and driven some companies to write off programs they once thought could be lucrative. AbbVie's announcement for Mavyret, approved in August to treat all hep C genotypes, only added to that pressure. After winning FDA approval, the Illinois drugmaker said its new therapy would be available at $13,200 per month—or $26,400 per treatment course—before discounts for most patients, significantly cheaper than already marketed options from Gilead Sciences, Merck and even AbbVie's own Viekira Pak. The drug is a combo of two new direct-acting antivirals, glecaprevir and pibrentasvir, and won FDA approval to treat adult hep C patients with genotype 1 through 6 who don’t have cirrhosis or who have mild cirrhosis, or those who are on dialysis. It’s the first pan-genotypic hep C drug with an eight-week treatment duration, according to the FDA. Other options take at least 12 weeks to cure the disease. — Eric Sagonowsky

33. Besponsa

Active ingredient: inotuzumab ozogamicin
Disease: relapsed or refractory acute lymphoblastic leukemia
Peak sales estimates: $1 billion to $2 billion
Approved: Aug. 17
Company: Pfizer

The scoop: Approved for the treatment of adults with relapsed or refractory acute lymphoblastic leukemia (ALL), Besponsa sped through regulatory review, thanks to breakthrough therapy and orphan drug designations. The FDA green-lighted the drug just weeks before it OK’d Novartis’ Kymriah, a CAR-T therapy for B-cell ALL, the first cell-based gene therapy for the disease. But Besponsa may have an advantage over Kymriah, at least in getting off the ground: The next-gen therapy uses a patient’s own stem cells, and so is complicated and expensive to produce. And while Besponsa does come with a boxed warning that it could lead to severe liver damage, both Pfizer and the FDA have said this approval is important for patients with few treatment options and low life expectancy. — Amirah Al Idrus

34. KedRAB

Active ingredient: human plasma-derived immunoglobulin
Disease: rabies
Peak sales estimates: N/A; market size in U.S. est. $100 million
Approved: Aug. 25
Companies: Kamada Ltd. and Kedrion Biopharma

Kamada and Kedrion Biopharma are no strangers to rabies. When KedRAB won U.S. approval in August, Kamada had already been selling the protein treatment in multiple countries under the brand name KamRAB, and Kedrion was an experienced supplier of high-titer rabies plasma. Designed to halt the rabies virus in patients who have already been exposed to it, KedRAB is cleared for use concurrently with a full course of rabies vaccine. The product will be launched by Kedrion in the U.S.—with a target of early 2018—under a development and marketing deal between the two companies. It’s Kamada’s second FDA-approved product after Glassia, which treats alpha-1 antitrypsin deficiency in patients with emphysema symptoms. — Tracy Staton

Vabomere

35. Vabomere

Active ingredients: meropenem and vaborbactam
Disease: complicated urinary tract infections
Peak sales estimate: $400 million
Approved: Aug. 29
Company: The Medicines Company

The scoop: The Medicines Company added to its modest product offerings in August with the FDA's approval of Vabomere to treat complicated urinary tract infections. Composed of the antibacterial meropenem and vaborbactam, which combats bacterial resistance, the drug launched in the U.S. in the fourth quarter. Vabomere works by addressing bacteria that produce the KPC enzyme, which make up the majority of carbapenem-resistant Enterobacteriaceae that the CDC has called an “urgent antimicrobial resistance threat.” Because of the drug's status as a Qualified Infectious Disease Product, The Medicines Company won an extra five years of exclusivity; Vabomere's patent protections stretch to 2031. Leerink Partners analysts have predicted the drug could haul in $400 million in sales by 2028. The company now has four marketed drugs in the U.S., according to its website: three in infectious diseases and one in cardiovascular care. — Eric Sagonowsky

36. Benznidazole

Active ingredient: benznidazole
Disease: Chagas disease
Peak sales estimate: N/A
Approved: Aug. 29
Company: Chemo Group, Mundo Sano and the Drugs for Neglected Diseases Initiative

The scoop: The partnership between Spanish multinational Chemo Group, its nonprofit foundation Mundo Sano and the Drugs for Neglected Diseases Initiative (DNDi) notched a major victory this summer when it won U.S. approval for benznidazole, a Chagas treatment that's already approved in eight Latin American countries. In doing so, the partners beat out Humanigen, which was formerly known as KaloBios and run by infamous pharma exec Martin Shkreli, to win a priority review voucher (PRV) that could be worth hundreds of millions of dollars. As part of the collaboration, the partnership committed 50% of the revenue from a PRV sale to a "far-reaching" access program to ensure benznidazole supply in the U.S. and elsewhere. The collaboration is working to improve on the "scandalous and unjustifiable" fact that only 1% of people living with the infectious disease can access treatment, according to DNDi Executive Director Bernard Pécoul, M.D. Chagas is spread by a parasite found in feces from the triatomine bug; about 300,000 people living in the U.S. have the disease. According to the company’s website, Chemo Group works to drive pharma prices down and develop treatments for neglected diseases. — Eric Sagonowsky

37. Kymriah

Active ingredient: tisagenlecleucel
Disease: B-cell acute lymphoblastic leukemia in children and young adults
Peak sales estimate: N/A
Approved: Aug. 30
Company: Novartis

If one drug would amount to the approval of the year, it would be Kymriah, the world’s first cell-based gene therapy. Novartis beat Kite Pharma to the punch—by a small margin, however—with its arrival at the finish line, and is now rolling out the treatment in U.S. medical centers.

The FDA itself called the approval “historic.” But Kymriah stands out in other ways as well. Because it uses a patient’s own cells to attack cancer, Kymriah is complicated and expensive to produce. T cells must be collected from each individual patient, genetically modified at a manufacturing facility, and then infused back into the patient. It’s also pricey at the patient level—$475,000 retail, for the small number of patients covered by its first indication, and that doesn’t include the required medical procedures associated with it.

So, even before Kymriah won approval, payers and market experts were calling for novel payment arrangements. Novartis struck a pay-for-performance deal with the U.S. government, a first; the company won’t accept payment if patients don’t respond within a month. The Swiss drugmaker also says it’s looking at indication-specific pricing; for diseases affecting a larger number of patients, the price tag would be lower. And Kymriah is already up for its second indication; Novartis filed an application for treating diffuse large B-cell lymphoma at the end of October. — Tracy Staton

38. Solosec

Active ingredient: secnidazole
Disease: bacterial vaginosis
Peak sales estimate: $100 million
Approved: Sept. 15
Company: Symbiomix Therapeutics

The scoop: Shortly after winning approval for Solosec in September, Symbiomix Therapeutics found itself the M&A target of India's Lupin, which shelled out $150 million to acquire the small company and expand its women's health business. Solosec won FDA approval on Sept. 15 as a single-dose oral treatment for bacterial vaginosis. According to the Newark, New Jersey-based biotech's approval announcement, the condition is the most common gynecologic infection in the U.S. and can be serious if improperly treated. Eager to expand its business in the area, Lupin quickly scooped the company up on Oct. 11. Numerous media reports said the company is expecting $100 million from the product if it can ramp sales up in the next three to four years. — Eric Sagonowsky

Aliqopa

39. Aliqopa

Active ingredient: copanlisib
Disease: relapsed follicular lymphoma
Peak sales estimate: $600 million
Approved: Sept. 14
Company: Bayer

The scoop: The FDA approved Aliqopa, Bayer’s first blood cancer drug, for the treatment of patients with relapsed follicular lymphoma who have received at least two prior treatments. The company is going all out with its blood cancer debut—it has hired hematology experts for its sales force and medical organization—in the hopes of outselling Gilead’s Zydelig, which won the FDA nod for blood cancers in 2014. Delivered via injection, Aliqopa may not be as convenient as Zydelig, a tablet, but Robert LaCaze, head of Bayer’s oncology strategic business unit, shrugged off the concern, saying it “allows the physicians to help better monitor the patients.” — Amirah Al Idrus

40. Verzenio

Active ingredient: abemaciclib
Disease: HR-positive, HER2-negative breast cancer
Peak sales estimate: $1.3 billion
Approved: Sept. 28
Company: Eli Lilly

The scoop: It’s not easy being third to market, but that’s the situation that Eli Lilly entered when Verzenio nabbed its FDA green light. The drug trailed behind Novartis’ Kisqali and, more importantly, Pfizer’s Ibrance—a blockbuster that had more than two years on the market alone before picking up any competitors. And as far as differentiators go, Verzenio has one that’s not so positive: It causes diarrhea in a high percentage of patients. As the Indianapolis pharma has insisted, though, that side effect is manageable—and there are good things that set Verzenio apart, too. Unlike its rivals, which can only be delivered in combo regimens, it can be used solo in some patients. Still, Lilly isn’t counting on its current indication alone to boost Verzenio to blockbuster status. On the expansion front, though, it hit its first snag in October, missing its endpoint in a phase 3 non-small cell lung cancer study. — Carly Helfand

41. Yescarta

Active ingredient: axicabtagene ciloleucel
Disease: relapsed or refractory large B-cell lymphoma
Peak sales estimate: $2 billion
Approved: Oct. 18
Companies: Kite Pharma, Gilead

The scoop: Two months after Gilead struck a $12 billion deal to acquire Kite Pharma, the pair struck gold: FDA approval of the latter’s Yescarta, a CAR-T therapy for relapsed or refractory large B-cell lymphoma, including aggressive non-Hodgkin lymphoma, in adults for whom two or more traditional treatments have failed. While the treatment did not work for all 101 patients in its pivotal trial, it represents hope for patients for whom chemotherapy has failed and who have no other options. Yescarta is the second treatment of its kind to get past the FDA, trailing the approval of Novartis’ Kymriah by nearly two months. Yescarta and Kymriah target two different types of blood cancer and carry different price tags: $373,000 and $475,000 respectively. But Gilead and Novartis may end up competing for market share in the future as they work to expand the indications for their treatments. — Amirah Al Idrus

42. Shingrix

Active ingredient: zoster vaccine recombinant, adjuvanted
Disease: shingles
Peak sales estimate: $1 billion
Approved: Oct. 20
Company: GlaxoSmithKline

The scoop: Industry watchers have expected a shingles vaccine market shake-up for some time now, and it came this year with GlaxoSmithKline's October approval for Shingrix, a vaccine analysts expect will grow to blockbuster sales by 2022. Shingrix has shown favorable efficacy in clinical testing, and right on the heels of approval, the CDC's Advisory Committee on Immunization Practices gave it a preferential vote over Merck's Zostavax. Shingrix is one of three products GSK is leaning on for growth as other areas of the business struggle. Ahead of the approval, the company has worked to build the case for Shingrix by touting data showing its vaccine offers lasting protection and can also protect those who’ve previously received Zostavax. — Eric Sagonowsky

43. Calquence

Active ingredient: acalabrutinib
Disease: mantle cell lymphoma
Peak sales estimate: N/A
Approved: Oct. 31
Company: AstraZeneca

The scoop: Back in 2015, AstraZeneca inked a $4 billion-plus deal with Acerta to gain access to Calquence—and that deal paid off with a Halloween go-ahead from regulators. With the green light, the British drugmaker stepped up to challenge AbbVie and Johnson & Johnson blockbuster Imbruvica in mantle cell lymphoma patients who have received at least one prior therapy. The way analysts see it, AZ has reason to be optimistic about the showdown, despite Imbruvica’s head start: In trials, Calquence posted a higher overall response rate than its rival did, though they cautioned that Imbruvica had a harder-to-treat cohort. In the price department though, Calquence has the distinct advantage. It bears a list price of $14,259 per month, compared with Imbruvica’s $14,804. Calquence’s label also lacks some specific warnings that Imbruvica’s includes, such as precautions related to hypertension and embryo-fetal toxicity. — Carly Helfand

44. Vyzulta

Active ingredient: latanoprostene bunod ophthalmic solution
Disease: open-angle glaucoma or ocular hypertension
Peak sales estimate: N/A
Approved: Nov. 2
Company: Valeant

The scoop: Picked up in Valeant’s $8.7 billion buyout of Bausch + Lomb in 2013, Vyzulta encountered a couple of hurdles before bagging FDA approval in November. The drug’s approval, for the reduction of eye pressure in open-angle glaucoma or ocular hypertension, was delayed thanks to two complete response letters linked to manufacturing problems at a Bausch + Lomb facility in Florida. Vyzulta was tagged to be a potential blockbuster, but former CEO Michael Pearson made the prediction before the manufacturing issues surfaced. However Vyzulta’s sales shake out though, Valeant may not be the company to collect, as new CEO Joseph Papa is said to be trying to sell off the eye-care unit in its quest for cash to pay down debt. — Amirah Al Idrus

45. Prevymis

Active ingredient: letermovir
Disease: to prevent infection after bone marrow transplant
Peak sales estimate: $370 million by 2020
Approved: Nov. 8
Company: Merck

The scoop: Designed to fight off cytomegalovirus infection in adults undergoing stem cell transplants, Merck’s Prevymis is the first new drug for CMV infection to get past the FDA in 15 years. While CMV is harmless to most people, it can be fatal for those who have received a bone marrow transplant. Given up to 100 days following transplant, Prevymis fends off infection and reactivation in patients who already carry the virus. Analysts anticipate the drug could rake in $370 million in sales by 2020—a modest 1% of Merck’s annual pharma revenue, but a boost that could help buoy a company whose anti-infective division recently took a few hits. Just this year, Merck ditched a three-drug hepatitis C combo and had to swallow a $2.9 billion write-down on a hep C drug it acquired in 2014. — Amirah Al Idrus

46. Fasenra

Active ingredient: benralizumab
Disease: severe asthma
Peak sales estimate: $2.1 billion in 2026
Approved: Nov. 14
Company: AstraZeneca

The scoop: When AstraZeneca’s Fasenra arrived on the scene in November, it was already the third product in its class to launch. But the therapy has some factors on its side that it hopes can help it make up ground. For one? Price. For the first year of treatment, Fasenra’s price before rebates and discounts will stand at $38,000—in line with its competitors’. After that, though, the annual price will dip to $28,000 or $33,000 for six or seven doses. Fasenra is also dosed less frequently than its nemeses, with patients receiving the drug every four weeks for the first three doses and every eight weeks after that; GlaxoSmithKline’s Nucala and Teva’s Cinqair are administered monthly. GSK, though, isn’t about to let its new rival run away with the market. The British drugmaker is “ready to fiercely compete,” Cheryl MacDiarmid, GSK’s SVP of primary care, said in a November interview. — Carly Helfand

47. Mepsevii

Active ingredient: vestronidase alfa-vjbk
Disease: mucopolysaccharidosis type VII
Peak sales estimate: $75 million
Approved: Nov. 15
Company: Ultragenyx

The scoop: Rare disease biotech Ultragenyx picked up its first FDA approval for Mepsevii in November, and while the product’s sales won’t make a splash in the larger biopharma industry, the drug does advance care for a group of patients who previously had no other options. Mepsevii, an enzyme replacement therapy, treats a lysosomal storage disorder called mucopolysaccharidosis type VII in children and adults. But due to a small patient pool—only 200 patients in the developed world have the disease—sales expectations are about $75 million at peak, according to one analyst. Ultragenyx said its drug will cost an average of $375,000 per patient per year after discounts. With the approval, the company also picked up a valuable FDA priority review voucher, one of which sold for $125 million in November. — Eric Sagonowsky

48. Hemlibra

Active ingredient: emicizumab-kxwh
Disease: hemophilia A
Peak sales estimate: $2 billion
Approved: Nov. 16
Company: Roche

The scoop: Ratcheting up the threat to Shire's hemophilia franchise, Roche won approval for blockbuster-to-be Hemlibra. Approved to prevent or reduce bleeding in adults or children with hemophilia A who have developed antibodies called factor VIII inhibitors, Hemlibra will carry a steep price tag: $482,000 for the first year and $448,000 for subsequent years. Roche garnered approval in a comparatively small patient pool initially but is testing the drug in a larger noninhibitor population that should bring larger sales over time, according to analysts. In the approved indication, Roche priced its drug at a steep discount to existing therapies. Market watchers are mixed on peak sales estimates, with consensus at $2 billion and Jefferies' team coming in much higher at $5 billion. The drug is expected to compete with Shire's hemophilia franchise, which that company picked up in its $32 billion Baxalta buyout. — Eric Sagonowsky

49. Ozempic

Active ingredient: semaglutide
Disease: Type 2 diabetes
Peak sales estimate: $2.7 billion
Approved: Dec. 5
Company: Novo Nordisk

The scoop: Diabetes drug giant Novo Nordisk has its next offering for the evolving field in Ozempic, a weekly GLP-1 treatment that's approved in addition to diet and exercise to improve glycemic control in adults with Type 2 diabetes. As Novo's follow-up to blockbuster Victoza, Ozempic is carrying big expectations at a pharma company that's been hit with diabetes pricing woes in recent years. Ozempic won the agency's backing based on clinical trial data showing it can reduce HbA1c levels compared to placebo, Merck's Januvia, AstraZeneca's Bydureon and Sanofi's Lantus. What's more, the drug also has favorable head-to-head data over Eli Lilly's Trulicity. Analysts with life science commercial intelligence firm Evaluate have predicted the drug will generate $2.7 billion in sales by 2022, representing one of the year's biggest launches. — Eric Sagonowsky

50. Xepi

Active ingredient: ozenoxacin
Disease: impetigo due to Staphylococcus aureus or Streptococcus pyogenes
Peak sales estimate: N/A
Approved: Dec. 11
Company: Medimetriks Pharmaceuticals

The scoop: After licensing U.S. rights to ozenoxacin from Spain's Ferrer back in 2014, Medimetriks Pharmaceuticals won approval for the topical cream in December. The FDA approved Xepi to treat the bacterial skin infection impetigo due to Staphylococcus aureus or Streptococcus pyogenes in patients 2 years and older. Fairfield, New Jersey-based dermatology drug company Medimetriks plans to launch the product in the first quarter of 2018, CEO Bradley Glassman said in a statement. Impetigo is contagious and frequently affects infants and young children, plus athletes or people living in enclosed environments. The condition accounts for 10% of skin issues in pediatric clinics, according to the drugmaker. — Eric Sagonowsky

51. Rhopressa

Active ingredient: netarsudil ophthalmic solution
Disease: glaucoma
Peak sales estimate: $443 million
Approved: Dec. 18
Company: Aerie Pharmaceuticals

The scoop: After boasting a workforce of just 94 employees at the end of last year, Aerie Pharmaceuticals will start 2018 by bulking up. In the wake of its Rhopressa approval in December, it's hiring 100 sales staffers as it preps a second-quarter launch. Rhopressa is FDA-approved to lower elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. In a statement, Aerie CEO Vicente Anido said the nod was the "single greatest achievement in Aerie's history to date," adding that it "represents exciting news for patients with open-angle glaucoma or ocular hypertension, and physicians." Canaccord Genuity predicts the drug will bring in $443 million by 2027, but a combo of the drug and the widely prescribed latanoprost could bring in $989 million that year, they said. Aerie plans to file for approval with the combo, dubbed Roclatan, in 2018's second quarter. — Eric Sagonowsky

52. Luxturna

Active ingredient: voretigene neparvovec-rzyl
Disease: RPE65 mutation-associated retinal dystrophy
Peak sales estimate: $381 million
Approved: Dec. 19
Company: Spark Therapeutics

The scoop: Spark Therapeutics may have won FDA approval for America's most expensive medicine, depending on the company's pricing announcement expected in January. In mid-December, the drugmaker's Luxturna passed FDA gatekeepers, becoming one of the world's first gene therapies; it treats an inherited form of vision loss that can result in blindness. The one-time treatment works by delivering a gene called RPE65 into a patient's retinal cells, which then produce a protein to restore vision loss. Spark didn't disclose its price right away, but analysts have said it could cost $1 million, a figure that pharma critics would likely jump on despite the advanced science involved. Evercore ISI analysts have predicted it will generate $381 million in 2019, a figure that falls to $158 million by 2024. — Eric Sagonowsky

53. Steglatro

Active ingredient: ertugliflozin
Disease: Type 2 diabetes
Peak sales estimate: $2 billion
Approved: Dec. 19
Companies: Pfizer, Merck

The scoop: With Steglatro, the fourth drug of its kind to hit the market, Pfizer and Merck have their work cut out for them. The other three SGLT2 drugs have enjoyed a decent head start over Steglatro, with Eli Lilly and Boehringer Ingelheim’s Jardiance and AstraZeneca’s Farxiga scoring approval in 2014, and J&J’s Invokana getting the OK in 2013. But Steglatro was approved both solo and as part of a pair of combo treatments: Steglujan, which will combine it with Merck’s Januvia, the leading DPP-4 drug, and Segluromet, which pairs it with metformin. The Steglujan combo could help Merck and Pfizer undercut its SGLT2 rivals while also keeping Januvia competitive. — Amirah Al Idrus

54. Macrilen

Active ingredient: macimorelin acetate
Disease: for the diagnosis of adult growth hormone deficiency
Peak sales estimate: $70 million
Approved: Dec. 20
Company: Aeterna Zentaris

The scoop: FDA approval for Macrilen, a ghrelin agonist for the diagnosis of patients with adult growth hormone deficiency, came after a bumpy road. It was delayed back in 2014 with a Complete Response Letter that called for another phase 3 trial, which concluded in January this year. The drug stimulates the pituitary gland to secrete growth hormone, which is measured in four blood samples collected over 90 minutes after administration. It is an alternative to the traditional insulin tolerance test, which involves multiple blood draws over a period of several hours. — Amirah Al Idrus

55. Giapreza

Active ingredient: angiotensin II
Disease: to treat dangerously low blood pressure in adults with shock
Peak sales estimate: $500 million
Approved: Dec. 21
Company: La Jolla Pharmaceutical Company

The scoop: La Jolla’s Giapreza squeaked in at the end of the year, making it the 46th new molecular entity to bag FDA approval in 2017. The drug is approved to increase blood pressure in adults with septic or other distributive shock. SunTrust analysts expect it to initially be used as a third-line treatment if norepinephrine and vasopressin prove ineffective. SunTrust’s Yatin Suneja wrote that it could see second-line use as well. — Amirah Al Idrus