Monday, July 15, 2024

The latest data presented at WMS 2023 confirm the enduring effectiveness of Nexviazyme® in treating Pompe disease

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Sanofi has presented new data at the 28th Annual Congress of the World Muscle Society (WMS), highlighting the continued efficacy of Nexviazyme® (avalglucosidase alfa) in treating Pompe disease. These findings, supported by nearly three years of data and real-world evidence encompassing various patient scenarios, reinforce the therapeutic value of Nexviazyme for individuals with Pompe disease. Nexviazyme is approved for late-onset Pompe disease in the U.S. and other markets, as well as for infantile-onset Pompe disease in Europe and other regions. Additionally, it is currently undergoing Phase 3 clinical trials in the U.S. for infantile-onset Pompe disease.

The data covers a wide spectrum of Pompe patients, including those with late-onset or infantile-onset Pompe disease, treatment-naïve individuals, and those who transitioned from the previous standard of care, alglucosidase alfa (known as Myozyme® or Lumizyme®). Key insights from the Phase 3 COMET trial indicate that Nexviazyme significantly improved or maintained respiratory function and mobility in patients, regardless of their treatment history. Moreover, patients who switched from alglucosidase alfa to Nexviazyme exhibited good tolerability.

Sanofi’s longstanding commitment to addressing rare diseases, particularly Pompe disease, led to the development of Myozyme in 2006, the first approved treatment for the condition. Subsequently, Nexviazyme was introduced to meet the evolving needs of the Pompe community. Pompe disease is characterized by low levels of the enzyme acid alpha-glucosidase (GAA), resulting in glycogen accumulation within muscle cells and potential damage to skeletal and cardiac muscles. The disease can manifest as infantile-onset Pompe disease (IOPD) or late-onset Pompe disease (LOPD), both of which can have severe consequences if left untreated.

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