Amicus Therapeutics has announced they have begun a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug combination, AT-GAA, to treat late onset Pompe disease (LOPD).

AT-GAA is the combination of cipaglucosidase alfa (ATB200) with miglustat. Cipaglucosidase alfa is a new enzyme replacement therapy. Miglustat is a drug approved to treat Gaucher disease in the United States and Niemann-Pick disease in Europe. According to Amicus, miglustat stabilizes the effectiveness of cipaglucosidase alfa.

A rolling BLA submission means that the company can submit portions of the application to the FDA when those portions are complete. To date, Amicus has submitted nonclinical data about cipaglucosidase alfa to the FDA and is in the process of submitting the chemistry, manufacturing and controls (CMC) component. The final clinical trial portion of the submission is expected to be submitted in the first half of 2021.

The clinical component of the submission will include data from Amicus’ ongoing Phase 1/2 (ATB200-02) and Phase 3 (ATB200-03, or PROPEL) studies.

The PROPEL study is a 52-week, double-blind randomized study comparing the efficacy, safety and tolerability of AT-GAA compared with alglucosidase alfa. The primary endpoint is the six-minute walk test. Results from this pivotal study are expected in the early 2021.

Since cipaglucosidase alfa is a recombinant enzyme and considered a biologic drug, it is filed with the FDA as a BLA instead of as a New Drug Application (NDA). Miglustat will be filed as an NDA. The review process is slightly different for an NDA and a BLA submission. The FDA will view an NDA submission to determine if a drug is safe and effective whereas a BLA submission is reviewed to determine if a drug is safe, pure, and potent.

Tagged: chaperone, clinical trials, FDA