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EMA accepts regulatory submission for avalglucosidase alfa.

First regulatory milestone

  • Avalglucosidase alfa, an investigational enzyme replacement therapy for patients with Pompe disease, reaches its first important regulatory milestone
  • Pompe disease affects an estimated 50,000 people worldwide
  • Submission based on positive data from two trials including both infantile-onset and late-onset Pompe disease patients
  • Regulatory approval in Europe anticipated in the second half of 2021
  • Milestone builds on company’s 20+ year commitment to the Pompe disease community
  • Avalglucosidase alfa receives Promising Innovative Medicine designation in UK, adding to U.S. Breakthrough Therapy designation received earlier this year

PARIS – October 2, 2020

The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.

Full appraisal questions

The MAA is based on positive data from two trials:

  • Phase 3, double-blind, comparator-controlled, pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease. Results from this trial were presented during a Sanofi-hosted virtual scientific session in June 2020.
  • Phase 2 mini-COMET trial, which evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infantile-onset Pompe disease previously treated with alglucosidase alfa. Results from this trial were presented at the WORLDSymposium, in February 2020.

Further information

 Go to Sanofi Genzyme website