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Plot twist as FDA U-turns on Amicus drug decision

Published on 11/07/17 at 02:49pm

The news continues to flow out of the FDA, as it grabs another headline by backtracking on its previous decision demanding further data for Amicus Therapeutics’ drug. The company should now be able to file an NDA in the 4th quarter, with an accelerated approval, for its drug Galafold.

This position change from the FDA means that the drug could reach the market by next year, after it had been predicted that it would need years to gather the extra data to satisfy the agency.

John Crowley, CEO of Amicus, was an already high-profile figure within biotech – after a Hollywood film had been made of his struggle to develop a treatment for children, who live with a rare condition called Pompe disease. The film itself had been based on a best-selling book.

The CEO’s story was further propelled into the spotlight as President Trump referenced Megan Crowley’s plight to suggest that more could be done to speed up drug approvals. Trump said, at a joint session of Congress that Crowley’s , “Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need”.

Amicus have been clear in its statement that the decision was reached purely through “discussions with and written communication from the FDA”, distancing the company from any suggestion that politics may have played a role.

With a case as high-profile as this, however, it seems unlikely that the influence of such a public episode of support did not influence the decision, even if in simply minor way. It is definitely an unusual step by the FDA to take such action that would always lead to such a discussion.

The drug in question, Galafold (migalastat), is an oral medicine for the treatment of Fabry disease.  It has already been approved by the European Commission for the disorder last year and has been in development for more than a decade.

“This guidance from the FDA marks a tremendous step forward for thousands of people living with Fabry disease in the United States,” stated John F. Crowley, CEO of Amicus Therapeutics. “We are moving ahead expeditiously with our NDA submission and accelerating the US pathway for migalastat. Today is a seminal moment in the development of migalastat and a testament to the dedication and perseverance of the patients, physicians and employees who have worked so hard on the development of this precision medicine.”

It is seminal moment for Galafold and an interesting development at the FDA, coming as it does shortly after a raft of new changes that the FDA has pushed through on generic drugs and some further decisive action, such as the decision to ask Endo to pull its opioid painkiller. There is a sense that the FDA is continuing to push the envelope under its new commissioner and it raises the question of what further action the agency will take to speed up the drug approval process.

Ben Hargreaves

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