FDA Approvals of 2012

Last year turned out to be a record-setter for new drug approvals in the U.S. The FDA blessed 39 new molecular entities (NMEs) in 2012, the most in 16 years and even more than the 35 racked up in 2011. And some experts are heralding a new era of productivity for biopharma R&D.

We'll see. Analysts at McKinsey predict that 35 approvals a year will become standard news in the industry through 2016. Compare that with the first decade of the new millennium, when an average of 24 drugs per year won FDA approval. It even beats out the 31-per-year average in the 1990s.

A new era of productivity would be--at the risk of stating the obvious--an extremely important development. The patent cliff has taken a toll on some of the industry's biggest drugs. Revenues continue to fall on those products to generic competition; governments are cranking back reimbursements; and development costs continue to rise. And while the worst of the patent cliff may be past for many companies, expirations will still take their toll. EvaluatePharma forecasts that $290 billion in sales are at risk from patent expirations between 2012 and 2018. Overall, branded drug sales were expected to fall 3.5% for 2012, according to a report from IBISWorld, and to recede about 2.6% a year for several years more. Jobs in the industry are expected to decline as sales do.

So, a stepped-up pace of new drug launches would give drugmakers more products and more sales to make up for their losses elsewhere. But is that predicted productivity a promise--or merely a hope? Development costs are way up, and pipelines are still lacking. Deloitte said last year that the cost of developing a drug had climbed 23% to more than $1 billion, while the average number of late-stage compounds in development fell to 18 from 23. And some of the industry's most anticipated products proved this year to be big disappointments, from the hepatitis C drug Bristol-Myers Squibb ($BMY) scrapped after patients died during a trial, to the very recent failure of Merck's ($MRK) cholesterol fighter Tredaptive, the third blood-lipid remedy it abandoned last year.

In fact, Tim Anderson at Bernstein Research recently reported that the Phase II success rate for experimental drugs cratered to 22% for the 5-year period ending in 2011. The success rate for the previous 5 years--2003 to 2007--amounted to 34%. Phase III was better, but that rate still fell to 65% from 70%. At the same time, the number of preclinical programs needed to produce a single new drug grew from 12 to 30.

Some experts point out that blunt numbers are only part of the picture. The big question is, how much money do all of these drugs generate? Despite its optimism about raw approval figures, McKinsey expects less on the sales side. The firm predicts "average peak-year sales of innovative products" will track down from about $900 million for the long list of products launched last year, to around $600 million for products launched in 2015.

It would be great if companies could generate more money from fewer drugs. But there are many signs that returns on investment will continue to get squeezed--and so the industry needs to not only bring costs down but also to generate even more new drugs to make up for lower per-product returns. The strategies for doing so are as varied as the drugs on this new approvals list.

The 2012 FDA approvals list runs the gamut. It includes everything from a new erectile dysfunction drug to a treatment for overactive bladder to one for weight-loss. There are nearly a dozen cancer drugs. There's one used in a test to rule out Alzheimer's disease, although none to actually treat that condition. And this is just a sampling.

There were only 4 companies that got more than one drug approved out of the 39. Forest Laboratories ($FRX), Roche's ($RHHBY) Genentech unit, and Sanofi ($SNY) got two FDA approvals in 2012. Industry leader Pfizer ($PFE), grabbed 5 of the approvals. 

As always, we welcome comments about what this list might say about the biopharma industry today--and about its future. -- Eric Palmer (email | Twitter)

Related Articles:
Special Report: Top 15 drug patent losses for 2013
New cancer drugs help spur drug approvals to 16-year high 
Deloitte: The price of R&D success soars as investment returns shrivel 
U.S. branded sales to end 2012 with 3.5% decline

Drug: Voraxaze
Generic molecule: glucarpidase
Company: BTG International
Approval date: Jan. 17

The scoop: The year's first drug approval went to the U.K.'s BTG for Voraxaze, a new treatment designed to reduce methotrexate concentrations in the plasma of patients receiving chemotherapy. The market is only estimated to be about $15 million.

Drug: Picato
Generic molecule: ingenol mebutate
Company: Leo Pharma
Approval date: Jan. 23

The scoop: Picato is a topical treatment for actinic keratosis (AK), a precancerous condition that can lead to squamous cell carcinoma (SCC), the second most common type of skin cancer. The topical gel treatment is completed within two or three days, compared with the weeks or even months other options require.

Drug: Inlyta
Generic molecule: axitinib
Company: Pfizer
Approval date: Jan. 27

The scoop: This twice-a-day pill represented another approval of a targeted cancer drug for Pfizer ($PFE) for treating kidney cancer. In 2011, Pfizer nabbed an FDA OK for its non-small cell lung cancer treatment Xalkori. Pfizer also markets two other drugs for advanced kidney cancer, Sutent and Torisel. Inlyta joined a crowded market and Pfizer is only forecast to earn about $500 million from the drug.

Drug: Erivedge
Generic molecule: vismodegib
Company: Genentech
Approval date: Jan. 30

The scoop: Developed with partner Curis ($CRIS), Roche's ($RHHBY) Genentech won a landmark approval for the targeted cancer drug. After collecting significant efficacy data from a Phase II study on basal cell carcinoma, the company headed straight to regulators for a decision. With no other treatments available for these patients, they won the approval well ahead of the agency's decision deadline. 

Drug: Kalydeco
Generic molecule: ivacaftor
Company: Vertex Pharmaceuticals
Approval date: Jan. 31

The scoop: Vertex Pharmaceuticals won early approval from the FDA for its drug Kalydeco, which is the first sanctioned drug that addresses the root cause of the disease. The drug is approved for CF patients with a specific mutation. Only about 1,200 patients in the U.S. have the G551D mutation, so like most drugs with such a small population, the pill comes with a hefty price tag--$294,000 a year. But Vertex is developing the drug in combination with other compounds, with the potential of treating way more CF patients.

Drug: Zioptan
Generic molecule: tafluprost
Company: Merck
Approval date: Feb. 10

The scoop: Merck ($MRK) says this is the first (get ready for a mouthful) preservative-free prostaglandin analog ophthalmic solution and is for treating elevated eye pressure in some patients with the most common form of glaucoma. Merck sells the ointment in the U.S. and most of Europe, while it licensed it to Japanese drugmaker Santen in Japan, Germany and northern Europe.

Drug: Surfaxin
Generic molecule: lucinactant
Company: Discovery Laboratories
Approval date: March 6

The scoop: It was a long, hard haul for the specialty drug company to get this one approved. The FDA several times asked the company for more data. After a rejection in 2009, when cash and patience were running short, the Discovery board tossed Robert Capetola, its CEO since the company's founding 13 years earlier, and for awhile replaced him with board Chairman Thomas Amick, a Johnson & Johnson ($JNJ) alum.

Drug: Omontys
Generic molecule: peginesatide
Company: Affymax
Approval date: March 27

The scoop: This anemia drug is the first competition for Amgen's ($AMGN) Epogen drug for kidney patients on dialysis, and analysts think it could capture a $700 million slice of that market by 2018. But to get it, Affymax will have to scramble. It has a marketing deal with Japan's Takeda and its drug is given once a month, compared with the three times a month for Epogen. But the two are priced similarly and Amgen has had two decades to establish loyalty. To that end it inked an exclusive, 7-year deal with the leading dialysis provider, DaVita, and a three-year, nonexclusive arrangement with Fresenius, which happens to have participated in clinical trials of Omontys.

Drug: Amyvid
Generic molecule: florbetapir F18
Company: Avid Radiopharmaceuticals
Approval date: April 6

The scoop: When Eli Lilly ($LLY) picked up Amyvid in its $800 million acquisition of Avid Radiopharmaceuticals, it surely planned to pair up this brain dye, which is used for ruling out Alzheimer's disease, with the treatment that it has been trying to move forward. But while it got a qualified approval for the dye, its two late-stage trials for Alzheimer's candidate solanezumab crashed and burned this summer. The test measures brain plaque buildup but the troublesome issues surrounding the potential for misdiagnosis led an expert committee as well as the FDA to reject Lilly's Amyvid early last year. Being careful not to oversell it at this point, Lilly says Amyvid is one tool docs can use to evaluate patients.

Drug: Stendra
Generic molecule: avanafil
Company: Vivus
Approval date: April 27

The scoop: The FDA issued word in April that it had stamped its OK on Stendra (avanafil), an erectile dysfunction treatment intended to boost blood flow to the penis before sex. The drug will enter a crowded field of rival and better-known treatments in a mass market, leaving some analysts to suggest that the Mountain View, CA-based biotech ($VVUS) would be better off making a deal on it as soon as possible with a Big Pharma company.

Drug: Elelyso
Generic molecule: taliglucerase alfa
Company: Pfizer
Approval date: May 1

The scoop: This orphan drug for Gaucher disease dropped into a market that has been owned by Genzyme and its Cerezyme. While not a large market, Genzyme has made money off of it and Pfizer and partner Protalix ($PLX) believe that they can as well. In the timing-is-everything sphere, it got to the market when Genzyme was only beginning to recover from several years of supply disruptions tied to a plant that had to be closed. Capitalizing on that, Pfizer not only priced Elelyso at a 25% discount to its competitor, it said it was setting up a system that would "endeavor" to maintain a 24-month supply, a clear dig at the Sanofi ($SNY) unit's problems. Israel's Protalix heralded the approval as an endorsement of its plant cell-based manufacturing system. Elelyso is made from carrot cells rather than the more complex mammalian cell-based system that Genzyme uses.

Drug: Perjeta
Generic molecule: pertuzumab
Company: Genentech
Approval date: June 8

The scoop: This new targeted breast cancer drug from Genentech is designed for use in tandem with the older Herceptin. Like Herceptin, it has blockbuster potential. Forecasts put Perjeta sales at $600 million to $1 billion initially and reaching as high as $8.5 billion if it gets approved for a couple of other indications. That compares with the more than $6 billion in sales Herceptin should pump out this year for Genentech parent Roche ($RHHBY).

Drug: Belviq
Generic molecule: lorcaserin hydrochloride
Company: Arena Pharmaceuticals
Approval date: June 27

The scoop: Belviq is the first weight-loss drug to be approved in more than a decade. It was a long time coming and with the approval the work is not over for Arena Pharmaceuticals ($ARNA). While some analysts see Belviq hitting $1 billion in sales, Arena has to convince doctors that drugs are the way to go in weight loss and payers that they should cover it, particularly since the drug has potential for abuse. Also the data for Belviq was not terribly compelling. The FDA found that patients lost 3% to 3.7% of their body weight and kept it off for a year. That amounts to just over 11 pounds max for a 300 pound person. Also, Belviq almost immediately faced competition from Vivus' ($VVUS) Qsymia.

Drug: Myrbetriq
Generic molecule: mirabegron
Company: Astellas Pharma
Approval date: June 28

The scoop: This new leaky bladder treatment from Japanese drugmaker Astellas Pharma will sell into a market in the U.S. where an estimated 33 million to 44 million suffer from overactive bladders. Myrbetriq goes up against Pfizer's Detrol, Warner Chilcott's ($WCRX) Enablex, and, of course, its own Vesicare, which generated $1 billion in sales last year but which loses its patent in 2018. The advantage of the once-daily Myrbetriq is that it helps without affecting patients ability to urinate, like some competing drugs.

Drug: Prepopik
Generic molecule: sodium picosulfate, magnesium oxide, citric acid
Company: Ferring Pharmaceuticals
Approval date: July 16

The scoop: Ferring's Prepopik is a bit of an oddball in the newly approved drug category because it has been available outside the U.S. since 1980. It is a low-volume, orange-flavored, dual-acting stimulant and osmotic laxative used for the admittedly unpleasant process of cleansing of the colon to get ready for a colonoscopy in adults. The company points out that "the sheer volume of prep solutions can prevent patients from adequately completing their prep regimens." Prepopik requires only 10 ounces and it is recommended that patients split the dose between the night before and day of, possibly getting better compliance.

Drug: Kyprolis
Generic molecule: carfilzomib
Company: Onyx Pharmaceuticals
Approval date: July 20

The scoop: Kyprolis is for the treatment of multiple myeloma patients who have already failed two therapies, making it a last-ditch option for many. It won accelerated approval from the FDA. And analysts think the drug, which comes with a price of about $10,000 a month, could generate upward of $1.5 billion a year in sales. Its approval also has sparked talk of a buyout of developer Onyx ($ONXX) with Bayer seen as a likely bidder.

Drug: Tudorza Pressair
Generic molecule: aclidinium bromide
Company: Forest Laboratories
Approval date: July 23

The scoop: The FDA approved this new lung drug, an inhaled treatment for chronic obstructive pulmonary disease, when Forest ($FRX) needed it most. Not only had it lost its patent for the blockbuster Lexapro, but the approval came right in the middle of the company's proxy fight with investor Carl Icahn. Analysts put sales for 2015 at about $250 million, however, filling only a part of the gap left by the newly off-patent antidepressant Lexapro.

Drug: Zaltrap
Generic molecule: ziv-aflibercept
Company: Sanofi
Approval date: Aug. 3

The scoop: This treatment for colorectal cancer may be remembered less for its efficacy than for the precedent Sanofi ($SNY) set by halving the price on the treatment, three months after it was approved. The company put it on fire sale after three doctors from Memorial Sloan-Kettering Cancer Center announced in The New York Times that their hospital wouldn't be using Zaltrap because it wasn't any better than existing treatments, but its $9,600 a month price tag made it more expensive, something they said just won't play in the new fiscal realities of healthcare. Sanofi responded by acknowledging "market resistance" and said it would discount the drug. The question is what effect it will have on the pricing of other drugs and whether with the discount Sanofi might actually pick up market share from the established treatment Avastin from Roche.

Drug: Stribild, also known as Quad
Generic molecules: elvitegravir, cobicistat, emtricitabine, tenofovir disoproxil fumarate
Company: Gilead Sciences
Approval date: Aug. 27

The scoop: EvaluatePharma says Stribild will be one of the most widely used anti-virals by 2018 and puts its worldwide sales at $2.7 billion by then, which puts Quad at 27 on its list of top 50 drugs by 2018. Upon approval, critics jumped on the price of the drug--$28,500. Healthcare advocates and AIDS activists were among them, but they were also accompanied by business and industry analysts.

Drug: Neutroval
Generic molecule: tbo-filgrastim
Company: Sicor Biotech, subsidiary of Teva Pharmaceuticals
Approval date: Aug. 29

The scoop: Sicor Biotech's tbo-filgrastim, which will be marketed by Teva ($TEVA) as Neutroval, is intended to reduce the time certain patients receiving cancer chemotherapy experience severe neutropenia, which is a decrease in neutrophils, white blood cells that fight infection. Tbo-filgrastim is intended for use in adults taking chemotherapy drugs that lead to a decrease in the production of these cells in the bone marrow; it stimulates production of neutrophils. Upon approval, Teva touted the drug as the first new granulocyte colony-stimulating factor (G-CSF) to be approved in the U.S. in more than a decade. Teva won't begin marketing the drug until November 2013 as part of an agreement with Amgen.

Drug: Linzess
Generic molecule: linaclotide
Company: Ironwood Pharmaceuticals
Approval date: Aug. 30

The scoop: The approval of partner Ironwood's linaclotide in late August is one of many reasons Forest Labs has been oft-cited as a takeover target in biopharma. Forest markets the drug, which is OK'd for chronic idiopathic constipation and to treat irritable bowel syndrome with constipation. Morgan Stanley has estimated potential peak sales at $2 billion.

Drug: Xtandi
Generic molecule: enzalutamide
Company: Medivation
Approval date: Aug. 31

The scoop: The drug won an OK a full three months ahead of the FDA's PDUFA date. Astellas is partnered on the program. With a yearly price tag of about $90,000, analysts say peak sales could range from $1 billion to $2 billion. It is approved for post-chemo, castration-resistant patients. But Medivation ($MDVN) hopes to win the same kind of upstream pre-chemo use that Zytiga from Johnson & Johnson just received, an indication that analysts say could double the drug's sales.

Drug: Bosulif
Generic molecule: bosutinib
Company: Pfizer
Approval date: Sept. 4

The scoop: Bosutinib is an orphan drug for treatment of chronic myelogenous leukemia. The FDA estimates that 5,430 Americans will be diagnosed with CML this year. Bosulif, a kinase inhibitor, became the third new cancer drug from Pfizer Oncology's pipeline to be approved by the FDA in a 13-month period. On the eve of approval, a spokesperson for Pfizer told FierceBiotech that the drug would cost less than $8,200 a month.

Drug: Choline C 11 Injection
Generic molecule: Choline
Organization: Mayo Clinic PET Radiochemistry Facility
Approval date: Sept. 12

The scoop: In September, the Mayo Clinic became the first facility approved to manufacture the Choline C 11 injection, a PET imaging agent used in detecting recurring prostate cancer. The injection helps produce an image that is useful in locating testing sites in the body for signs of cancer. Although PET imaging is not considered a replacement for tissue sampling and testing, but it may reveal signs that cancer has returned when other more conventional imaging doesn't.

Drug: Aubagio
Generic molecule: teriflunomide
Company: Sanofi
Approval date: Sept. 12

The scoop: Soon after the news hit the wires that Sanofi ($SNY) had won a badly needed FDA approval for its oral multiple sclerosis drug Aubagio (teriflunomide), a rep for Sanofi subsidiary Genzyme began touting the price advantages. At $45,000 a year, the drug will be trying to grab market share from Copaxone, Avonex and Gilenya. Genzyme is also seeking approval of Lemtrada for MS, where it's been given a good shot, and if approved will contribute to the company's growing MS franchise.

Drug: Stivarga
Generic molecule: regorafenib
Company: Bayer HealthCare
Approval date: Sept. 27

The scoop: FDA approved regorafenib just three months after it was granted fast-track review status and more than a month ahead of the drug's PDUFA date. The drug treats metastatic colorectal cancer, and proved enticingly effective in Phase III studies. Analysts expect sales of the drug to reach $1.25 billion. Onyx Pharmaceuticals, Bayer's partner on the drug, gained $160 million upfront and a 20% royalty stream from Bayer, after filing a lawsuit with the company over ownership rights.

Drug: Jetrea
Generic molecule: ocriplasmin
Company: ThromboGenics
Approval date: Oct. 18

The scoop: The approval of ocriplasmin marked the first time a drug has been available for treatment of symptomatic vitreomacular adhesion, which is currently treated surgically. The company experienced strong growth in 2012, in part due to an FDA panel recommendation for approval of ocriplasmin in July. Peak sales are estimated at about $900 million, Reuters reported.

Drug: Fycompa
Generic molecule: perampanel
Company: Eisai
Approval date: Oct. 22

The scoop: Eisai's Fycompa is a glutamate receptor antagonist and the first of its kind designed to treat epilepsy. The drug is a blockbuster hopeful for the Japanese drugmaker, and analysts have projected it could bring in $500 million in sales by 2015 and eventually break the $1 billion barrier.

Drug: Synribo
Generic molecule: omacetaxine mepesuccinate
Company: Teva Pharmaceutical Industries
Approval date: Oct. 26

The scoop: Teva ($TEVA) got the FDA's OK for Synribo to treat chronic myelogenous leukemia (CML) patients whose cancer has remained after taking two or more previous tyrosine kinase inhibitors (TKIs). Synribo became the second drug the agency approved to treat this form of leukemia in as many months, after the FDA's nod for Pfizer's Bosulif in September.

Drug: Xeljanz
Generic molecule: tofacitinib
Company: Pfizer
Approval date: Nov. 6

The scoop: The approval of Pfizer's ($PFE) oral RA drug couldn't have come at a better time for the Big Pharma company, which has been struggling recently with its late-stage programs. Analysts projected that peak sales of the drug could reach $2 billion to $3 billion a year. A Pfizer spokesperson told Bloomberg that the drug will cost $2,055 a month, or a bit more than $24,000 a year.

Drug: Cometriq
Generic molecule: cabozantinib
Company: Exelixis
Approval date: Nov. 29

The scoop: The multikinase inhibitor's approval to treat thyroid cancer made it the second drug approved to treat the medullary form of the disease in as many years; AstraZeneca's ($AZN) Calpresa was approved in November 2011. FierceBiotech featured cabozantinib as one of the top 10 cancer drugs in late-stage development earlier this year. Exelixis ($EXEL) has been looking for approval for the much larger prostate cancer market, but the drug in still in Phase III trials to treat the castrate-resistant form of the disease.

Drug: Raxibacumab
Generic molecule: raxibacumab
Company: GlaxoSmithKline (via Human Genome Sciences)
Approval date: Dec. 14

The scoop: The FDA's approval of the raxibacumab injection marked the first monoclonal antibody approved under the FDA's Animal Efficacy Rule, which allows findings from animal trials when it's not possible or ethical to conduct human studies, as in this case. Raxibacumab is approved to treat inhalational anthrax which is caused by breathing in spores of the bacterium Bacillus anthracis. The drug works by neutralizing the toxins that can lead to tissue damage and death.

Drug: Iclusig
Generic molecule: ponatinib
Company: Ariad Pharmaceuticals
Approval Date: Dec. 14

The scoop: The FDA announced it had approved ponatinib to treat two types of leukemia a full three months before the drug's PDUFA date. The drug, selected as a blockbuster hopeful by analysts, will be sold at a wholesale acquisition cost of $9,580 a month, or $115,000 a year, according to CEO Harvey Berger in a call with analysts following approval. FierceBiotech featured ponatinib as one of the 10 cancer drugs in late-stage development to watch this year.

Drug: Signifor
Generic molecule: pasireotide diaspartate
Company: Novartis
Approval date: Dec. 14

The scoop: The FDA's approval of injectable pasireotide diaspartate to treat the underlying mechanism causing Cushing's disease provides a new option for patients suffering from the disease, which until now has been treated by surgery.

Drug: Gattex
Generic molecule: teduglutide
Company: NPS Pharmaceuticals
Approval date: Dec. 21

The scoop: The FDA approved NPS' Gattex, a GLP-2 drug, for treating short bowel syndrome just as the holiday exodus began. NPS prepared for the transition to the commercial realm this year; CEO Francois Nader told FierceBiotech that 2012 would be transformational for his company. The U.S. regulator's approval of the drug follows the European Commission's approval in August.

Drug: Juxtapid
Generic molecule: lomitapide
Company: Aegerion Pharmaceuticals
Approval date: Dec. 24

The scoop: The FDA approved lomitapide at a crucial point for Aegerion ($AEGR); the company has been slowly growing its relatively small commerical operation and focuses its resources on this program alone. Aegerion will market the drug for patients suffering from the rare genetic disease homozygous familial hypercholesterolemia which can lead to fatal heart conditions; the drug will include a boxed warning of liver toxicity.

Drug: Eliquis
Generic molecule: apixaban
Companies: Pfizer, Bristol-Myers Squibb
Approval date: Dec. 28

The scoop: Days before the year's end, FDA approved Eliquis--and 3 months before the drug's PDUFA date. The joint venture by Bristol-Myers Squibb ($BMY) and Pfizer ($PFE) is a blood thinner meant for reducing risk of stroke and blood clots in patients with non-valvular atrial fibrillation. Twice-delayed by the FDA, Eliquis's approval is predated by two other warfarin alternatives: Johnson & Johnson's Xarelto and Boehringer Ingelheim's Pradaxa.

Drug: Fulyzaq
Generic molecule: Crofelemer
Company: Salix Pharmaceuticals
Approval date: December 31

The scoop: The FDA approved Salix Pharmaceutical's diarrhea drug Fulyzaq for HIV patients on the last day of the year. Derived from the Croton lechleri plant, Fulyzaq is the second botanical drug ever approved by the FDA. Although not expected to be a big earner for Salix with an estimate of only $33 million in 2014, the company ($SLXP) counts on Fulyzaq to broaden its product line.

Drug: Sirturo
Generic molecule: bedaquiline
Company: Johnson & Johnson
Approval date: December 31

The scoop: The FDA made history on the last day of 2012 as the agency approved for the first time in forty years a new treatment for tuberculosis. From Johnson & Johnson's ($JNJ) Janssen Therapeutics unit, Sirturo was approved for use in patients with "multidrug resistant pulmonary tuberculosis (TB) when other alternatives are not available." Given a fast-track designation despite a warning concerning potentially fatal abnormal hearth rhythms, Sirturo is considered a "much-needed treatment for patients who don't have other therapeutic options available."