Avalglucosidase Alfa: First Approval

Version 2 2021-09-07, 22:16

Version 1 2021-09-07, 21:49

online resource

posted on 2021-09-07, 22:16 authored by Sohita Dhillon

Declarations

Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Sohita Dhillon is a contracted employee of Adis International Ltd/Springer Nature and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable.

Additional information about this Adis Drug Review can be found here

Abstract

Avalglucosidase alfa (NEXVIAZYME™; avalglucosidase alfa-ngpt) is a hydrolytic lysosomal glycogen-specific recombinant human α-glucosidase product being developed by Sanofi Genzyme (formerly Genzyme Corporation) for the treatment of Pompe disease. Pompe disease is an autosomal recessive lysosomal storage disease caused by a deficiency of the lysosomal enzyme acid α-glucosidase (GAA), which results in intralysosomal accumulation of glycogen in various tissues. In August 2021, avalglucosidase alfa received its first approval in the USA for the treatment of patients 1 year of age and older with late-onset Pompe disease (GAA deficiency). In July 2021, avalglucosidase alfa received a positive opinion in the EU for long-term enzyme replacement therapy for the treatment of patients with Pompe disease. The drug is under regulatory review in the UK and Japan, and clinical studies are underway in several countries worldwide. This article summarizes the milestones in the development of avalglucosidase alfa leading to this first approval for late-onset Pompe disease.