New ERT Nexviazyme approved for treatment of Pompe disease

May 15, 2024

healthcaretoday, Pompe disease, genetic disorder, Nexviazyme, IOPD, LOPD, muscle function, rare disease, SANOFI,

Pompe disease is caused by a deficiency in acid glucosidase which primarily affects the muscles, and patients experience symptoms like muscle weakness, breathing difficulties and heart problems.

Nexviazyme (Avalglucosidase alfa) is the new enzyme replacement therapy (ERT) approved in Malaysia for the treatment of Pompe Disease, a rare and progressive genetic disorder that is often fatal if left untreated.

Pompe disease is caused by a deficiency in acid glucosidase (GAA), which leads to an accumulation of lysosomal glycogen in the body’s tissues. This primarily affects the muscles, and patients experience symptoms like muscle weakness, breathing difficulties and heart problems that may require the use of wheelchairs and respiratory support. While it can affect individuals of all ages, Pompe disease is broadly divided into Infantile Onset Pompe Disease (IOPD) and Late Onset Pompe Disease (LOPD).

Improving lung function, walking distance, and muscle strength
“Nexviazyme offers patients an effective treatment option and relief from the symptoms of Pompe disease by helping to improve lung function and walking distance. There is also evidence that treatment with Nexviazyme helps improve muscle strength,” said Quek Wee Ling, General Manager, Specialty Care (Thailand, Malaysia, Singapore and Vietnam), Sanofi.

Nexviazyme is approved for long-term enzyme replacement therapy for the treatment of patients with Pompe disease, delivered intravenously every two weeks. Clinical studies noted improved motor function among patients with IOPD who had shown suboptimal response to earlier treatment, while patients with LOPD experienced improved respiratory muscle function, measured using Forced Vital Capacity (FVC), and increased ability to walk further.

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