Amicus maps out a 3-year delay at the FDA for lead drug, sending shares into a tailspin

CEO John Crowley

CEO John Crowley

More than a year after Amicus Therapeutics was forced to do a sudden about-face and walk back promises of a quick submission for its lead drug migalastat, the biotech has laid out plans for a new trial that will be needed to win an FDA approval — pushing back any prospect of an OK for treating Fabry disease by at least three more years.

The Cranbury, NJ-based biotech’s shares $FOLD cratered on the news, dropping 28%.

The biotech says it will now need to mount a special trial to explore the drug’s impact on GI symptoms. It will execute a 12-month, randomized study with 35 patients, with data arriving in 2019.

The latest setback for a company that has been on a roller coaster ride for years dashed hopes of investors that the biotech would be able to line up for an accelerated approval of the drug — already OK’d in Europe — with the data already in hand. Investigators had pinned down evidence of migalastat’s success in reducing levels of GL-3 that accumulates in the kidney cells of patients with the disease. The drug is designed to work among patients with amenable mutations covering about half of the population, roughly 25% of whom are in the US.

Genzyme and Shire market infused enzyme replacement therapies for the condition, which Amicus wants to replace with their pill.

Going all the way back to the fall of 2015, Amicus execs had initially expressed enthusiasm for their sit-down with regulators at the FDA. Then they took a second look at the minutes of their meeting and radically changed course, raising the prospect of a new trial.

CEO John Crowley put the delay in the best light possible.

“While we believe that the totality of the data from our studies with migalastat support the submission of a new drug application today, we acknowledge the FDA’s position that accelerated approval based on kidney GL-3 reduction is not currently an option. We have thus defined a plan to collect additional GI data to support full approval for migalastat that we believe is feasible in a reasonable amount of time and with a high likelihood of success based on positive GI data generated in our previous Phase III Study 011. FDA has been flexible in allowing a crossover design and in our use of established GI endpoints to measure clinical benefit in Fabry patients. We are fully committed to the additional work necessary to move migalastat toward approval in the United States.”

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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