CHMP Adopts Positive Opinion for Sanofi’s Avalglucosidase Alfa as a Next-Generation Therapy for Pompe Disease
Sanofi
Pharmaceutical R&D Developer
Committee for Medicinal Products for Human Use
Committee for Medicinal Products for Human Use (CHMP)The Committee for Medicinal Products for Human Use (CHMP) is the committee within the European Medicines Agency (EMA) responsible for human medicines. The CHMP replaced the former Committee for Proprietary Medicinal Products (CPMP) in May 2004.The CHMP plays a vital role in the authorization of medicines in the European Union (EU). In the centralized procedure, the CHMP is responsible for: 1) conducting initial assessments of marketing authorization applications across the EU; assessing modifications or extensions to existing marketing authorizations (“variations”); considering recommendations from the Agency’s Pharmacovigilance Risk Assessment Committee regarding the safety of medicines on the market, and, where necessary, advising the European Commission to amend the marketing authorization of a medicinal product, or to suspend or withdraw it from the market.The CHMP also evaluates medicines authorized at the national level that are referred to the EMA, with the aim of maintaining a harmonized position throughout the EU.Furthermore, the CHMP and its working groups promote the development of medicines and pharmaceutical regulation by: providing scientific advice to companies researching and developing new medicines; developing scientific and regulatory guidelines to assist pharmaceutical companies in preparing marketing authorization applications for human medicines; and collaborating with international partners to harmonize regulatory requirements.
Recently, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) announced a positive opinion on avalglucosidase alfa, developed by Sanofi, supporting its use for the treatment of patients with glycogen storage disease type II (also known as Pompe disease).
Pompe disease is a rare degenerative muscle disease that affects an individual's mobility and respiratory function. It is caused by a genetic defect or dysfunction of lysosomal acid α-glucosidase (GAA), leading to glycogen accumulation in muscle cells throughout the body. This ultimately results in irreversible muscle damage, including to the respiratory muscles and diaphragm that support lung function, as well as other skeletal muscles that affect mobility.
Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of GAA to muscle cells. To reduce glycogen accumulation, GAA must be delivered to the lysosomes within muscle cells. Research led by Sanofi has primarily focused on enhancing the delivery of GAA to muscle cell lysosomes by targeting the mannose-6-phosphate (M6P) receptor, which plays a critical role in GAA transport. Compared with the standard therapy alglucosidase alfa, avalglucosidase alfa features approximately 15-fold higher M6P content, designed to help improve cellular uptake of the enzyme and enhance targeted glycogen clearance. If approved, it would provide a potential new standard of care for these patients. The U.S. FDA is currently reviewing the regulatory application for avalglucosidase alfa, with a decision expected in August this year.
▲ Impaired glycogen degradation in patients with Pompe disease (Image source: pompe.com)
The clinical trials supporting the approval of avalglucosidase alfa include a pivotal Phase 3 clinical trial evaluating the safety and efficacy of avalglucosidase alfa compared with alglucosidase alfa (standard of care) in patients with late-onset Pompe disease, as well as results from a Phase 2 clinical trial conducted in patients with infantile-onset Pompe disease who had previously been treated with alglucosidase alfa.
References:
[1] Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 19-22 July 2021. Retrieved July 25, 2021, from https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-19-22-july-2021
*Disclaimer: This article was written by a contributing author to Sina Medical News. The views expressed are solely those of the author and do not represent the position of Sina Medical News.▽Follow 【WuXi AppTecGermany】WeChat Official Account
