Regulatory

Once rejected, FDA now rolls out a short red carpet for Amicus’ migalastat

John Crowley

Amicus CEO $FOLD John Crowley must have made a very favorable impression on the FDA as he lobbied senior officials to overturn their rejection of his experimental Fabry drug migalastat.

Not only did regulators just accept the filing for a new review, the agency is giving it priority status, cutting the rethink to 6 months and flagging a clear indication that it’s an odds-on favorite for an official marketing OK .

Cutting 4 months off the review process, the FDA will have its new decision out by August 13.

Analysts are already counting the near-term revenue that Amicus stands to earn. Joseph Schwartz at Leerink noted:

With the PDUFA goal date of Aug 13, 2018, US revenue could contribute to FY2018 revenue guidance of $75M-$85M announced last month. We continue to see strong growth drivers for Amicus this yr. as the company continues to advance its migalastat (Galafold; Fabry disease) and ATB200/AT2221 (Pompe disease) programs through the clinic.

Crowley had employed some dubious positions as he fought against the rejection, which came with a demand from the FDA for a new Phase III study of gastrointestinal symptoms. Initially, the CEO had told investors it would take 2 years to come up with the data. But he told Janet Woodcock and others that the actual timeline would be 5 to 7 years, which made the development program impractical.

Scott Gottlieb

This was one of three drug programs that the FDA reversed itself on in the months after Scott Gottlieb took over as commissioner at the FDA, with Eli Lilly and TherapeuticsMD — chaired by ex-HHS secretary Tommy Thompson — getting a do-over. The agency has never explained the rejections, never explained why it decided to reconsider their decision, or why it would offer Amicus a quick decision now.

But they never do, required by the rules to stay quiet. Gottlieb had initially said he would press for the redacted publication of complete response letters like these, but he recently shrugged that off.

“Congratulations, again!” Crowley wrote in a note to Gottlieb attached to his case for a review. “As discussed, the hopes and well being of so many living with rare, devastating diseases rests now with your great leadership and wisdom. Thanks for your attention to the attached.”

Gottlieb’s staff told me that the commissioner never saw that packet, redirecting it to other senior officials at the FDA. Officials, though, declined to explain what Crowley meant by the word “again,” or if Gottlieb had discussed the issue with the CEO.

That note, along with Crowley’s assertions on the timeline to respond, were obtained under FOIA by the Project on Government Oversight.

Gottlieb has openly advocated for a more efficient review process, particularly where rare disease are concerned. That also squares with President Donald Trump’s vow to speed up the regulatory process. And after starting out with a high rating from the industry, biotech execs are now cheering him on as one of the best things that’s happened at the FDA in years.


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