New Data Supports the Effectiveness of Nexviazyme for Pompe Disease Treatment
Paris, October 6, 2023 – Sanofi, a leading pharmaceutical company, has presented new data at the 28th Annual Congress of the World Muscle Society (WMS) in Charleston, South Carolina, reinforcing the efficacy of Nexviazyme® (avalglucosidase alfa) for the treatment of Pompe disease. These findings build upon the extensive body of evidence supporting the use of Nexviazyme in various clinical situations for both late-onset and infantile-onset Pompe disease patients.
Nexviazyme is a monotherapy that has received approvals in the United States and other markets for late-onset Pompe disease treatment, as well as for infantile-onset Pompe disease in Europe and other countries. Additionally, Nexviazyme is currently undergoing Phase 3 clinical trials for the treatment of infantile-onset Pompe disease in the U.S.
The data shared at WMS encompassed research involving patients with late-onset or infantile-onset Pompe disease, including those who were treatment-naïve or had transitioned from a previous standard of care, alglucosidase alfa, marketed under the names Myozyme® and Lumizyme®. The studies also included patients with diverse baseline characteristics.
Of particular significance are the results from the Phase 3 COMET trial, which examined the long-term efficacy, safety, and durability of Nexviazyme in individuals with late-onset Pompe disease. The data collected over nearly three years demonstrate that patients who initiated treatment with Nexviazyme, regardless of whether they were treatment-naïve or had previously received treatment, experienced improvements or stabilization in respiratory function and mobility. Furthermore, the data indicate that Nexviazyme was well tolerated by patients who switched from alglucosidase alfa, the previous standard of care.
The data being presented at WMS reinforce the existing evidence supporting the use of Nexviazyme in the treatment of Pompe disease. With positive results observed in various patient populations, we remain confident in the value of Nexviazyme as a compelling treatment option, said a spokesperson from Sanofi.
Sanofi has a longstanding commitment to developing treatments for rare diseases, including Pompe disease, spanning over 20 years. They initially developed Myozyme, the first approved treatment for Pompe disease in 2006. Subsequently, Sanofi has collaborated closely with the Pompe community to address unmet patient needs, resulting in the development of Nexviazyme.
Pompe disease is characterized by low levels of the enzyme acid alpha-glucosidase (GAA), leading to glycogen accumulation in muscle cells throughout the body. This buildup can cause irreversible damage to skeletal and cardiac muscles. The disease can manifest as infantile-onset Pompe disease (IOPD), the most severe form with rapid onset in infancy, or as late-onset Pompe disease (LOPD), which progressively damages muscles over time. Without treatment, IOPD can lead to heart failure and death within the first year, while LOPD patients may require mechanical ventilation or a wheelchair as the disease progresses.
The newly presented data at the WMS highlight the efficacy and safety of Nexviazyme in treating Pompe disease. Sanofi’s ongoing dedication to improving the lives of individuals with this condition underscores the importance of continued research and development in rare disease therapeutics. With Nexviazyme demonstrating positive outcomes in multiple patient cohorts, it remains a promising option for people living with Pompe disease.
