Muscular Dystrophy Association Celebrates FDA Approval of (Nexviazyme) for Treatment of Pompe disease

NEW YORK, Aug. 6, 2021 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant accelerated marketing approval to avalglucosidase alfa (Nexviazyme) for the treatment of people 1 year of age and older living with late-onset Pompe disease. It is the second approved drug to treat Pompe disease. Nexviazyme will be made available in the United States and marketed by Sanofi Genzyme.

Myozyme (alglucosidase alfa), also developed by Sanofi Genzyme, was approved by the FDA in 2006 for individuals with infantile-onset Pompe disease, and was later marketed as Lumizyme (alglucosidase alfa) for individuals with late-onset Pompe disease. In November 2020, avalglucosidase alfa received FDA Breakthrough Therapy, Priority Review, and Fast Track designations based on positive data from two trials in patients with late-onset and infantile-onset Pompe disease, respectively.

"The approval of Nexviazyme provides another option for patients with Pompe disease," says MDA's Executive Vice President and Chief Research Officer Sharon Hesterlee, PhD. "MDA funded the foundational work at Duke University that contributed to the development of the first drug approved for Pompe, Myozyme, and it's gratifying to see the evolution of new therapies for this disease.  There is always more work to be done to better understand the disease burden for the community and to develop additional therapies and MDA will continue to stand by the Pompe community in support these efforts." 

Pompe disease is a rare degenerative muscle disorder that affects approximately 3,500 people in the US. Pompe disease results from mutations in the gene encoding the acid alpha-glucosidase (GAA) enzyme, which plays a role in the body's ability to break down the complex sugar glycogen. With insufficient GAA, glycogen builds up and damages muscle cells, particularly in the heart and skeletal muscles. This can lead to muscle weakness and premature death from respiratory or heart failure. Enzyme replacement therapy, such as Lumizyme or Myozyme, is the only effective treatment available for Pompe disease. ERT delivers a man-made version of the GAA enzyme into the body via injection. Nexviazyme is a second-generation ERT designed to improve GAA uptake by specific tissues in the body and more effectively achieve clearance of glycogen.

Clinical trials support approval of Nexviazyme

The FDA based its decision to grant accelerated marketing approval to Nexviazyme on positive results from the phase 3 COMET trial, which is expected to conclude by September 2024, and the phase 2 mini-COMET trial, which is expected to conclude in December 2024. COMET data showed meaningful improvements in respiratory muscle function and mobility in patients treated with avalglucosidase alfa, demonstrating comparable efficacy to Lumizyme treatment. For more information about the Comet and mini-COMET trials, visit ClinicalTrials.gov and enter "NCT02782741" or "NCT03019406" into the search box, respectively.

To learn more about the approval of Nexviazyme read the FDA press release.

MDA resources

Since its inception, MDA has invested more than $5 million in Pompe disease research.

MDA's Resource Center provides support, guidance, and resources for patients and families, including information about the approval of Nexviazyme, open clinical trials, and other services. Contact the MDA Resource Center at 1-833-ASK-MDA1 or ResourceCenter@mdausa.org.

About the Muscular Dystrophy Association 
For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. MDA Advocacy supports equal access for our community, and each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted at mda.org/COVID19. For more information, visit mda.org.

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SOURCE Muscular Dystrophy Association