Posted by Michael Wonder on 02 Oct 2020
EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease
2 October 2020 - Submission based on positive data from two trials including both infantile-onset and late-onset Pompe disease patients.
The EMA has accepted for review the marketing authorisation application for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).
Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.
