Sanofi's Next-Generation Pompe Disease Therapy Avalglucosidase Alfa Granted Clinical Trial Approval in China for Infantile-Onset Patients

By | Pharma Insights

According to the latest announcement on the official website of the Center for Drug Evaluation (CDE) under China's National Medical Products Administration (NMPA), Sanofi’s Class 1 new drug avalglucosidase alfa has received implied approval for clinical trials. It is intended to be developed as an enzyme replacement therapy for patients with early-onset Pompe disease. This investigational enzyme replacement therapy was recently approved in the United States for the treatment of late-onset Pompe disease. Previously, it had been granted Breakthrough Therapy designation, Fast Track designation, and Priority Review designation by the FDA for the treatment of Pompe disease.

Screenshot source: CDE Official Website

Pompe disease is a progressive, debilitating muscle disorder that impairs mobility and respiratory function. The disease is caused by a genetic deficiency or dysfunction of lysosomal acid alpha-glucosidase (GAA). The resulting GAA deficiency leads to glycogen accumulation in muscle cells throughout the body, causing irreversible damage to various muscles, including the diaphragm, which supports respiratory function, and skeletal muscles that affect mobility, functional endurance, and breathing.

To reduce glycogen accumulation, GAA must be delivered to the lysosomes within muscle cells. The key pathway for transporting the GAA enzyme to intracellular lysosomes is the mannose-6-phosphate (M6P) receptor.

Avalglucosidase alfa, developed by Sanofi, targets the M6P receptor to enhance the delivery of GAA into the lysosomes of muscle cells. Studies indicate that, compared with the standard-of-care α-glucosidase, this product has an approximately 15-fold increase in M6P content, which is designed to improve cellular uptake of the enzyme and enhance targeted glycogen clearance.

In August 2021, the FDA announced the approval of this enzyme replacement therapy (brand name: Nexviazyme) for the treatment of patients aged 1 year and older with late-onset Pompe disease. The approval was based on positive results from the pivotal Phase 3 COMET trial, with data demonstrating that avalglucosidase alfa improves walking distance and respiratory function parameters in patients.

Sanofi stated in a press release that avalglucosidase alfa is a potential new standard of care for patients with late-onset Pompe disease. We hope this therapy will be approved in China soon, bringing new treatment options to more patients with Pompe disease.

References:

[1] Center for Drug Evaluation, National Medical Products Administration (CDE). Retrieved Sep 6, 2021, from http://www.cde.org.cn/news.do?method=changePage&pageName=service&frameStr=20

[2] FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease. Retrieved August 6, 2021, from https://www.globenewswire.com/news-release/2021/08/06/2276588/0/en/FDA-approves-Nexviazyme-avalglucosidase-alfa-ngpt-an-important-new-treatment-option-for-late-onset-Pompe-disease.html

[3] Kishnani P S , Attarian S , Borges J L , et al. Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients[J]. Molecular Genetics and Metabolism, 2021, 132(2):S57.

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