- The Food and Drug Administration has for a second time this year delayed its review of Amicus Therapeutics’ experimental treatment for Pompe disease, deferring action until the agency can inspect the drug’s manufacturing facilities in China.
- In a statement Friday, Amicus said travel restrictions related to COVID-19 prevented the FDA from conducting required inspections of its partner Wuxi Biologics’ manufacturing site in China. It was the only reason given by the FDA for the deferred action on the company’s application, according to Amicus.
- The company still expects the FDA to approve the two components of its therapy, called AT-GAA, for the rare muscle disorder. “We continue to believe this is a question of ‘when’ not ‘if’ AT-GAA will be approved” in the U.S., said Amicus CEO Bradley Campbell in the company’s statement.
Other approval applications have faced similar delays since COVID restrictions began in early 2020. But for Amicus, this one comes after years of challenges securing approval of its treatment as well as achieving profitability.
Initially, the company had hoped to pursue an accelerated approval for its therapy. But, in 2018, the FDA closed off that path to Amicus, saying more clinical data was needed. The company was required to begin a Phase 3 study that evaluated whether AT-GAA helped restore muscle function based on a 6-minute walk test compared with enzyme replacement therapy, the current standard of care.
After Amicus reached full enrollment of 123 patients in the trial, the FDA in early 2020 agreed to a rolling review of the company’s application. Earlier this year, the agency extended its review period to give it more time to review data from Amicus.
Pompe disease is an inherited disorder caused by a deficiency of the enzyme acid alpha-glucosidase, or GAA. Amicus is seeking approval of its treatment, a recombinant version of the enzyme cipaglucosidase alfa that is modified to improve its uptake into cells and is administered alongside a stabilizer molecule called miglustat.
The FDA gave no timeline for further action after its latest delay, Amicus said. The drug is also up for review in the European Union, which does not require a pre-approval inspection, and an opinion from its key regulatory committee is expected by the end of the year, Campbell said.
Amicus earlier this year abandoned plans to spin off its gene therapy business after a deal with a special purpose acquisition company unraveled, forcing it to lay off staff and cut costs to meet its goal of turning a profit in 2023. The company has one approved drug, Galafold, an oral treatment for the rare genetic disorder Fabry disease.
Amicus reported product sales of nearly $81 million in the second quarter — up by 4% on the year, all of it from Galafold — but reported a net loss of $62 million.
Amicus released its statement after Friday’s market close. Its shares were trading down by more than 2% Monday morning