Amicus Therapeutics (NASDAQ:FOLD) has announced that the commencement of the rolling Biologics License Application submission to the US FDA for its experimental two-component therapy, AT-GAA, for late-onset Pompe disease treatment.
Amicus to submit parts of BLA as they complete
It is important to note that the rolling BLA submission will allow Amicus to submit parts of the agency’s regulatory application as they complete them. This is instead of waiting to complete all sections of the BLA before submitting the entire BLA for review. At the beginning of this year, the FDA authorized the company to go on with the Rolling BLA submission.
AT-GAA is the company’s investigational therapy consisting of cipaglucosidase alfa (ATB200) administered in combination with miglustat (AT2221). ATB200 is a distinctive enzyme replacement therapy with enhanced carbohydrate structures, while AT2221 is an orally administered cipaglucosidase alfa stabilizer. The company has already submitted the nonclinical cipaglucosidase alfa BLA component, and it is on course for submission of the chemistry, manufacturing, and controls component. Amicus will submit the final component in 1H 2021. The use of cipaglucosidase alfa in conjunction with miglustat will be a separate NDA that the company will submit when it submits the rest of the cipaglucosidase alfa BLA.
BLA submission takes Amicus closer to delivering Pompe diseases treatment
Amicus CEO and Chairman John Crowley said that the BLA submission is a huge milestone for the company and the Pompe community. Crowley said that the rolling BLA for the experimental therapy supports the company’s belief in Pompe diseases’ huge unmet need and supports the strategy for AT-GAA advancement. He affirmed that the announcement and previously approved designations, including growing clinical data support the company’s belief that AT-GAA could potentially become a Standard of Care for Pompe patients.
The company received Breakthrough Therapy Designation from the FDA for AT-GAA to treat late-onset Pompe disease based on phase 1/2 clinical study efficacy results. The company expects to release topline results of the global Phase 3 PROPEL clinical trial of AT-GAA ion LOPD in Q1 2021.