FDA approves Sanofi drug for rare muscle disorder Pompe disease

FRANCE – Sanofi’s treatment for muscle disorder Pompe disease has received the U.S. health regulator’s nod, strengthening the French drug maker’s hold on the market for drugs to treat the rare but potentially fatal disease.

The U.S. Food and Drug Administration allowed Nexviazyme, an enzyme replacement therapy administered by injecting into a vein, to be used in patients aged at least a year, and above with late onset of Pompe disease.

Sanofi’s other enzyme replacement therapy for Pompe disease, Myozyme, was approved by the FDA in 2006, and Lumizyme in 2010 for late-onset of the disease and is used for all patients regardless of their age.

The disease, which affects 1 in 40,000 people in the United States, is characterized by a buildup of a complex sugar called glycogen in skeletal and heart muscles that could lead to muscle weakness and premature death.

Amicus Therapeutics (FOLD.O) is also developing a treatment for Pompe disease and in February reported that its experimental two-component therapy, AT-GAA, did not meet the main goal of statistical significance for superiority in a 6-minute walk test in a late-stage trial.

Sanofi’s Nexviazyme showed improvement in respiratory function and walking distance measures in people with late-onset of Pompe disease in its late-stage trial.

The company did not give a price for Nexviazyme, only said it will price it the same as alglucosidase alfa, sold under brand names Lumizyme and Myozyme.

The approval was widely celebrated by the Muscular Dystrophy Association (MDA). It is the second approved drug to treat Pompe disease for people 1 year of age and older living with late-onset Pompe disease.

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.

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