Fabry disease is caused by mutations of the GLA gene. Amicus asked the FDA to approve Galafold for Fabry patients older than 16 with mutations that can respond to migalastat. Up to half of the 1,000 or so Fabry mutations are in this category. Mutations that can respond are known as amenable mutations.
The European Union and a number of other countries have already authorized Galafold. Amicus is asking U.S. regulators to approve it as an alternative to intravenous enzyme replacement therapy.
In Fabry disease, a defective alpha-galactosidase A enzyme leads to the build-up of globotriaosylceramide lipids throughout the body, including the kidneys. Lipids include fats, oils and waxes.
Galafold restores the enzyme’s activity in people carrying amenable GLA mutations, who account for 35 to 50 percent of Fabry patients worldwide.
The FDA has granted orphan drug and fast track designations to Galafold. Both are aimed at speeding up a treatment’s development and regulatory review.
Without priority review, the FDA would have to decide by August 13, 2018, whether to approve Galafold. Priority review should lead to a quicker decision, which could making the drug available to American patients sooner.
“The FDA’s acceptance of our first Amicus NDA [New Drug Application] submission under priority review is an important step toward a potential oral precision medicine option for the Fabry disease community in the U.S.,” John F. Crowley, Amicus’s chairman and chief executive officer, said in a press release.
“With more than a decade of experience in treating patients with migalastat globally, our team at Amicus has collaborated with leading Fabry disease experts and patient organization leaders to assemble a robust NDA that emphasizes the breadth of our clinical [trial] data and experience delivering migalastat to patients,” Crowley said.
The New Drug Application for Galafold that Amicus submitted to the FDA is based on the results of a number of trials, including two pivotal Phase 3 studies. The FACETS trial (NCT01218659) involved Fabry patients who had never been treated for the disease. The ATTRACT trial (NCT01218659) covered patients who had been on enzyme replacement therapy.
EU regulators used the trial results to approve Galafold in 2016. Switzerland, Israel, Australia, Canada and South Korea have also approved it.
Spain’s national health system recently agreed to cover Galafold. And earlier this year Canada’s Drug Expert Committee recommended that the national health system cover Galafold as a long-term treatment for adults with Fabry disease who have an amenable mutation.
Meanwhile, Amicus is recruiting patients worldwide to participate in its expanded access program (NCT01476163). Under this program, doctors can ask Amicus to make the treatment available to patients for six months, with a chance to renew for another six months.
Up to 20 patients will be selected. More information about the program can be found here.