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2017 drug approvals: Records, milestones, and controversies

2017 drug approvals: Records, milestones, and controversies

In his first year as FDA Commissioner, Scott Gottlieb has chaperoned the agency through a blockbuster 2017 with 46 novel drug approvals to date. The final tally is the highest seen this century, surpassing the previous record holder 2015, which clocked in at 45. Both were more than double 2016’s sad tally of 22 new molecular entities (NMEs) approved.

While NMEs are overseen by the FDA’s Center for Drug Evaluation and Research (CDER), gene therapies fall under the jurisdiction of The Center for Biologics Evaluation and Research (CBER). As a result, three of the biggest approvals of the year came on top of the record-setting 46: Luxturna, a gene therapy for an inherited retinal disease, and the two CAR-T therapies Kymriah and Yescarta.

But it wasn’t all hugs and celebration. A number of approvals were met with significant controversy, due to a perceived lack of progress and/or sky-high pricing. Here’s a recap of some of the flashpoints for that debate.

Emflaza (deflazacort) for Duchenne muscular dystrophy

Marathon Pharmaceuticals had barely announced its new drug approval before the chaos erupted. While it had shown efficacy, Emflaza is a corticosteroid — part of a class of drugs that are already widely used for Duchenne muscular dystrophy (DMD). The exact same drug had been available overseas for decades at a cost of around $0.60 per dose. Marathon slapped an $89,000 price tag on its U.S. version. After much outcry from the media – and a harshly worded letter from a couple of politicians – the company quietly offloaded the drug.

Radicava (edaravone) for amyotrophic lateral sclerosis (ALS)

This approval caught a lot of people by surprise. FDA took the unusual measure of approving the drug based on a six-month study in Japan. Additional clinical trials in the United States were bypassed, seemingly to get the drug into patients’ hands. While it was an unusual approach, there was ample justification. ALS is a rapidly progressive neurodegenerative disease and a majority of patients die within two to five years of diagnosis. Radicava became the second-ever drug approved for ALS and the first in over 20 years.

Nerlynx (neratinib) for the treatment of HER2-positive breast cancer

Puma Biotechnology polarized the field and FDA committees alike with its submission for Nerlynx, a drug designed to lower the risk of a patient’s breast cancer recurring after treatment with trastuzumab (Herceptin). As such, it’s prophylactic: A majority of women wouldn’t have experienced a recurrence either way. In fact, if 100 women were treated for two years, just two would be spared cancer as a result of taking Puma’s drug — and almost all would experience side effects. In one study 96 percent of patients in the active arm had some degree of diarrhea and 40 percent of patients had Grade 3 diarrhea, which “may require treatment in the hospital or clinic.” Put together, the case for its approval was far from clear-cut.

Ocrevus (ocrelizumab) for relapsing and primary progressive forms of multiple sclerosis

Roche’s Ocrevus is undeniably effective, but it too came with some controversy. It’s an updated version of Rituxan (rituximab), first approved in 1997. The difference is that Ocrevus is a fully humanized monoclonal antibody and it costs $55,000 more per year than Rituxan. However, the biggest ethical issue isn’t the price. As reported, Rituxan could have been approved for multiple sclerosis (MS) years ago. By starting the approval process again with Ocrevus, many MS patients may have missed out.

So there you have it; 2017 was a record-setting year, but it was a mixed bag of approvals.

And in case you were wondering, 1,590 new molecular entities have now been approved in the history of the United States, the bulk of them since 1938 when the modern FDA was formed. (Check out this Drug Discovery Today paper for more of the backstory.)

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