Sanofi's New Pompe Disease Therapy Avalglucosidase Alfa (Nexviazyme) Approved in China

On October 7, SanofiSanofi announces its Elaprase® (generic name: idursulfase alfa) for injection (Nai Er Zan) Approved by the National Medical Products AdministrationAbout to be launched for the 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 genetic defect or dysfunction of acid alpha-glucosidase (GAA) and lysosomal glycogen accumulation, leading to the accumulation of glycogen in skeletal and cardiac muscle cells, which in turn causes muscle weakness and can even result in premature death due to respiratory failure or heart failure.

Avalglucosidase alfa is a highly efficient 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.2In August 2021, the productFDA Approval ObtainedApprovalCity,For the treatment ofFor the treatment of late-onset Pompe disease in patients 1 year of age and older;2022YearJuneApproved for marketing in Europe.

A randomized, double-blind, multinational, multicenter pivotal Phase III COMET study evaluated 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: during both the primary analysis period (49 weeks) and the extension study period, patients receiving...Patients treated with Avalglucosidase alfa showed a 2.65 (1.05) percentage point increase in the predicted percentage of Forced Vital Capacity (FVC).6-Minute Walk Test (6MWT）Distance increased by 18.60 (12.01) meters;During the extension study, patients who switched from first-generation enzyme replacement therapy to avalglucosidase alfa treatment showed an increase of 0.36 (1.12) percentage points in the predicted FVC percentage.6MWT distance increased by 4.56 (12.44) meters.

In addition,InDuring the preliminary analysis period,Avalglucosidase alfaThe safety profile was good, 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 in a prominent position.For reprinting, please leave a message or send a message to the WeChat Official Account backend, and indicate the name and ID of the official account.

↑ Long pressScan the code, Schedule Live ↑