Amicus rolling U.S. AT-GAA marketing application for Pompe disease

 

• Amicus Therapeutics (NASDAQ:FOLD) has initiated rolling marketing application to the FDA for AT-GAA for the treatment of late onset Pompe disease (LOPD), an inherited metabolic disorder caused by the buildup of glycogen in the body's cells due to mutations in the gene that codes for an enzyme called acid alpha-glucosidase, leading to organ dysfunction.
• The rolling submission allows submission of portions of the regulatory application as they are completed, rather than waiting until every section of the application is complete.
• Earlier this year, the FDA authorized Amicus to proceed with a rolling BLA submission.
• AT-GAA consists of cipaglucosidase alfa (ATB200), an enzyme replacement therapy with optimized carbohydrate structures, administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.
• Amicus has submitted the nonclinical component of the cipaglucosidase alfa application and is on-track to submit the chemistry, manufacturing and controls component, and the final clinical module in 1H of 2021.
• The company will separately submit miglustat application at the same time as the remainder of the cipaglucosidase alfa BLA.
• AT-GAA received FDA Breakthrough status for LOPD. Top-line data from ongoing Phase 3 PROPEL study of AT-GAA in LOPD is on-track for Q1 of 2021.
• Recently, under the priority review status, the FDA accepted  Sanofi's application for long-term enzyme replacement therapy for Pompe disease. Agency's action date is May 18, 2021.
• https://seekingalpha.com/news/3640312-amicus-launches-rolling-u-s-gaa-marketing-application-for-pompe-disease