After FDA About-Face, Amicus Plans NDA for Fabry Disease Candidate Migalastat quoting : GEN

Amicus Therapeutics said today it plans to submit an NDA for its Fabry disease candidate migalastat in the fourth quarter after the agency, in an unusual about-face, reversed a decision made last year to require an additional Phase III study assessing the drug in gastrointestinal symptoms. It takes now orphan drugs as much time to complete Phase III studies (the stage of drug development where efficacy is established) as it does for nonorphans," Crowley asserted. "Our slow and burdensome approval process at the FDA keeps too many advances, like the one that saved Megan's life, from reaching those in need," Trump told Congress. Amicus gained national attention earlier this year when President Donald Trump wove into his February 28 address to Congress the story of the daughter of the company's CEO. Trump related how the life of 20-year-old Megan Crowley, a Notre Dame sophomore, was saved through a Pompe disease treatment developed by a drug developer founded by her father John F. Crowley, now Amicus' chairman and CEO.




Amicus currently holds European approval for its Fabry Disease drug migalastat, marketed as Galafold. Today, Amicus Therapeutics (FOLD) has announced that the FDA will allow the company to submit an NDA for its drug migalastat that treats patients with Fabry disease. The U.S. market compromises about 30% of the total Fabry Disease population. Phase 3 DataThere was extremely positive results in patients with Gastrointestinal --GI --symptoms that have Fabry disease. Phase 3 data in Fabry Disease should be enough for FDA approval and that will give patients another treatment option.

FDA Reverses Decision, Allowing Amicus To File For Rare Disease Drug

Crowley is famous in biotech circles for his work developing a drug for Pompe disease, another rare disease from which his daughter, Megan, suffers. But Amicus still has to go through the process of approval, and the FDA could still reject the drug. Today, Amicus says that the FDA is reversing the decision, and that there is a clear path to approval. Amicus hopes to file a new drug application by the end of the year, and approval would take six months after that. In Fabry disease, a substance called GL-3 builds up in the body, causing pain, kidney failure, heart disease and stroke.


collected by :Lucy William

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