FDA to Decide on AT-GAA for Late-onset Pompe Around Mid-2022
The U.S. Food and Drug Administration (FDA) is reviewing applications from Amicus Therapeutics asking for the approval of AT-GAA as a treatment for late-onset Pompe disease (LOPD).
Amicus initiated a rolling application for AT-GAA late last year, which allowed the company to submit portions of its application as they became ready, instead of a waiting until all data was available as is standard.
The company now announced that it completed two applications covering each component of the two-part investigational therapy: a biologics license application (BLA) for cipaglucosidase alfa — an optimized form of the enzyme that is missing in Pompe disease — and a new drug application (NDA) for miglustat, a pharmacological “chaperone” that helps to stabilize the enzyme.
The FDA is expected to make a final decision on the NDA for miglustat by May 29. A decision on the BLA for cipaglucosidase alfa is expected by July 29.
“The FDA’s acceptance of these filings is an immensely important step forward for people living with Pompe disease and their families in the United States. Patients need new medicines as soon as possible,” John F. Crowley, chairman and CEO of Amicus, said in a press release.
“With today’s announcement, we remain confident in the potential of this medicine to become the next standard of care in Pompe disease,” Crowley added.
Amicus’ applications are supported by results from the Phase 1/2 ATB200-02 study (NCT02675465), as well as the Phase 3 PROPEL (NCT03729362) trial and its ongoing extension study. Results from ABT200-02 showed that a two-year treatment with AT-GAA improved muscle strength, motor function, and lung function in adults with Pompe disease.
Top-line data from PROPEL were announced earlier this year. The study enrolled 123 adults with Pompe disease, making it the largest placebo-controlled trial ever conducted in this disease. Results showed that the investigational therapy significantly improved physical and lung function.
Of note, most patients in these trials had previously been treated with the enzyme replacement therapy alglucosidase alfa (marketed as Lumizyme in the U.S.), the current standard Pompe treatment. In PROPEL, those who switched from alglucosidase alfa to AT-GAA were especially likely to show physical and lung function gains.
“We will work with great urgency with the FDA as they review the applications over the course of the coming months,” Crowley said, adding that Amicus is also “diligently working towards additional regulatory submissions outside of the U.S.”
A submission to the European Union is expected before the end of this year. Recently, AT-GAA was made available to select patients in the U.K. through an early access program, based on a positive opinion from a regulatory agency in that country.
Amicus is sponsoring two other ongoing clinical trials testing AT-GAA. The Phase 3 ZIP study (NCT03911505) is currently enrolling children and adolescents with LOPD at locations in the U.S., Canada, Japan, and Taiwan.
The Phase 3 and open-label Rossella trial (NCT04808505) is testing AT-GAA in about 22 children, from birth to 17 years old, with classic infantile-onset Pompe disease. The trial is presently recruiting participants at the University of Florida Clinical Research Center.