By Clifton Leaf and Sy Mukherjee
May 9, 2017

Good morning, readers. This is Sy.

The Food and Drug Administration (FDA) has been on a streak of late. Over the weekend, the agency approved the first new treatment for ALS, or Lou Gehrig’s disease, since 1995 (Mitsubishi Tanabe Pharma’s Radicava). The brings the total number of new medicine approvals up to 20 so far this year.

So how does that stack up against the FDA’s historical performance? Consider this: there were 22 new treatments cleared for the market in all of 2016.

To be fair, last year’s sluggish pace was a bit of an outlier compared to the near-record 2014 and 2015, when the FDA green lit 45 and 41 therapies, respectively. But this year’s specific approvals are striking for another reason: several of them are the first-ever drugs to be made available in the U.S. for certain diseases.

That includes: BioMarin’s Brineura for a specific form of the rare disorder Batten disease (and a treatment that, at a $700,000 annual list price, is one of the most expensive therapies in the world); Roche/Genentech’s Ocrevus, the first medicine cleared to treat more severe kinds of multiple sclerosis (MS); and Neurocrine Biosciences’ Ingrezza, the first drug for the neurological disorder tardive dyskinesia.

While many of these therapies are breakthroughs for patients who have very limited options, they also reflect a trend in drug development: an overwhelming focus on specialty cancer and rare disease drugs. Given the reality of opportunity costs, that might mean some other therapeutic spaces are getting short shrift.

Read on for the day’s news.

Sy Mukherjee


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