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. That will shave three or four years off the drug’s path to market if all goes well.
In Fabry disease, a substance called GL-3 builds up in the body, causing pain, kidney failure, heart disease and stroke. About 3,000 Americans have been diagnosed with the condition, according to Amicus. Migalastat is thought to work only in about half the patients with the disease, whose disorder is linked to certain genetic mutations.
The drug was approved in the European Union a year ago, but the FDA was declining to consider Amicus’ application, in part because one of two clinical trials of the drug had failed to meet its main prespecified goal. But John Crowley, Amicus’ chief executive, says that the company convinced the FDA to look at its data using new analyses, including some that had been asked for by European regulators, and patient testimony. “We didn’t ask them to lower the bar,” he says. “We just asked them to review the existing data.”
Crowley is famous in biotech circles for his work developing a drug for Pompe disease, another rare disease from which his daughter, Megan, suffers. The story was the subject of a book, The Cure, and a movie, Extraordinary Measures, which starred Harrison Ford. Earlier this year, President Donald Trump highlighted Megan Crowley’s story in a speech to Congress as an example of the need for a more flexible FDA.
But Crowley says he doesn’t think Trump’s speech had any impact on the FDA’s decision. “No, I didn’t get that sense at all,” he says. “This was a series of discussions on science.” He says that the tenor of the discussions with the FDA did not change when Scott Gottlieb, the current FDA commissioner, was approved. He also says he doesn’t think the controversial approval of Exondys 51, a drug from Sarepta Therapeutics for muscular dystrophy, set any precedent for his medicine, saying that there is much more data on migalastat. He does argue, however, that there is “an emerging framework at the FDA for rare diseases.” He notes the approval of BioMarin’s Bineura for Batten’s disease and an expanded approval for Vertex’s Kalydeco for cystic fibrosis as examples.
Crowley says that it is “very clear, very unambiguous” that Amicus can file with the FDA. The FDA was given a chance to review the company’s press release and had no objections or comments, he says. But Amicus still has to go through the process of approval, and the FDA could still reject the drug.