Despite a February adcom meeting urging the agency to wait for more data, the FDA has approved Karyopharm Therapeutics Inc.'s selinexor, in combination with dexamethasone (dex), as a new treatment for certain adults with relapsed refractory multiple myeloma (MM). The approval covers patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents and an anti-CD38 monoclonal antibody.
The FDA placed a clinical hold Wednesday on a phase I trial by Unum Therapeutics Inc. after a patient experienced serious adverse events that included grade 3 neurotoxicity and cytomegalovirus infection, and grade 4 respiratory distress.
With regulatory science always lagging innovation, ambiguity has long been a certainty at the FDA and is likely to become even more so as the pace of new technologies quickens.
In the words of former FDA Commissioner Scott Gottlieb, the five-week government shutdown that ended Jan. 25 was "the most difficult operational challenge we have faced in modern times." While the full impact of that challenge could ripple through the FDA for a while, it made little difference in the number of warning letters the agency sent out between Dec. 23 and Jan. 25.
Approval of Alexion Pharmaceutical Inc.'s Soliris (eculizumab) injection to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive makes it the first and only FDA-approved treatment. It's also one of the most expensive treatments in the world, making it vulnerable to off-label use and the eventual creep of biosimilars into the market.
Life has a lot of unknowns, and one of the big unknowns for 2019 is what the FDA will look like now that Scott Gottlieb has returned to the American Enterprise Institute. Norm Sharpless of the National Cancer Institute has been named the acting FDA commissioner – which by itself doesn’t mean a whole lot, other than that he has to be considered a lead candidate for the job – but the difficulty in knowing where the agency would go in terms of policy under...
Now that the dust is still swirling over the news that Scott Gottlieb will leave the FDA, it’s time to conduct a hasty post-mortem on his tenure at the agency, or perhaps more to the point, his lack of tenure. While it’s tempting to frame the question that way, it might be more salient to ask about the nature of the FDA commissioner’s job and whether it is still sufficiently politically insulated to do what is asked of it. Gottlieb had been on the job...
