Improve standard treatment EU CHMP supports the launch of Sanofi's new generation of Pompe disease therapy

A few days ago, the European Medicines Agency (EMA) Committee for Medicines for Human Use (CHMP) announced that it has issued a positive opinion on avalglucosidase alfa developed by Sanofi and supports its use in the treatment of glycogen storage disease type II (also known as Pang Shell disease) patients

Pompe disease is a rare degenerative muscle disease that affects an individual's ability to move and breathe

Avalglucosidase alfa is an enzyme replacement therapy under investigation designed to improve the delivery of GAA to muscle cells

▲Defects in glycogen degradation in Pompe disease patients (picture source: pompe.

The clinical trials supporting the approval of avalglucosidase alfa include a pivotal phase 3 clinical trial to evaluate the safety and efficacy of avalglucosidase alfa compared with α-glucosidase (standard treatment) in patients with late-onset Pompe disease

Reference materials:

[1] Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 19-22 July 2021.