Sanofi's Avalglucosidase Alfa (Nexviazyme) Approved in China for Long-Term Enzyme Replacement Therapy in Pompe Disease

On October 7, Sanofi announced that its injectable Avalglucosidase alfa (brand name: Nexviazyme) was approved for marketing by the National Medical Products Administration, for long-term enzyme replacement therapy in patients with Pompe disease (acid alpha-glucosidase [GAA] deficiency).

Pompe disease is a rare progressive neuromuscular disorder caused by a hereditary defect or dysfunction of acid alpha-glucosidase (GAA) and lysosomal glycogen accumulation, leading to the accumulation of glycogen in skeletal and cardiac muscle cells, resulting in muscle weakness and potentially premature death due to respiratory or heart failure.

Avalglucosidase alfa is a highly effective enzyme replacement therapy that targets the mannose-6-phosphate (M6P) receptor, thereby improving the delivery of GAA to muscle cells, allowing excess glycogen to be broken down and reducing damage to patients' muscle cells. In August 2021, the product received FDA approval for marketing to treat late-onset Pompe disease in patients aged one year and older; it was approved for marketing in Europe in June 2022.

A Randomized, Double-blind, Multinational, Multicenter Pivotal Phase III COMET Study Evaluating the Efficacy and Safety of Avalglucosidase Alfa in 100 Patients with Pompe Disease. Results from the 97-week extension study showed that, compared to baseline: patients receiving avalglucosidase alfa during both the initial analysis period (49 weeks) and the extension study period had an increase of 2.65 (1.05) percentage points in the predicted percentage of Forced Vital Capacity (FVC), and an improvement of 18.60 (12.01) meters in the 6-Minute Walk Test (6MWT) distance; patients who switched from first-generation enzyme replacement therapy to avalglucosidase alfa during the extension study experienced an increase of 0.36 (1.12) percentage points in the predicted FVC percentage, and an improvement of 4.56 (12.44) meters in 6MWT distance.

In addition, during the initial analysis period, Avalglucosidase alfa showed good safety, and no new safety events were observed during the extension study period.

Copyright © 2023 PHARMCUBE. All Rights Reserved.

Welcome to forward, share, and reasonably cite. When citing, please clearly indicate the source of the article; if you need to reprint, please leave a message to the WeChat Official Account or send a message, and include the name and ID of the official account.

Disclaimer: The information in this WeChat article is for general reference only and cannot be directly used as decision-making content. PharmaCube assumes no responsibility for any loss incurred by any party due to the use of the content herein.

Highlights

Online Live Streaming & Offline Salon