Posted by Michael Wonder on 18 Nov 2020
FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
18 November 2020 - Regulatory submission based on positive data from two trials in patients with late-onset and infantile-onset Pompe disease, respectively.
The U.S. FDA has accepted for priority review the biologics license application for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).
The target action date for the FDA decision is 18 May 2021.
