FDA Approves Sanofi's Xenpozyme for ASMD Treatment

The U.S. Food and Drug Administration (FDA) has approved Sanofi's Xenpozyme (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adults and pediatric patients. This approval marks Xenpozyme as the first and currently the only approved therapy specifically for ASMD.

ASMD, previously known as Niemann-Pick disease types A, A/B, and B, is an extremely rare and progressive genetic disease associated with significant morbidity and mortality. Symptoms can include an enlarged spleen or liver, breathing difficulties, lung infections, and unusual bruising or bleeding. Prior to this approval, ASMD management relied on supportive care for individual symptoms and careful monitoring.

Sanofi has focused on bringing hope to patients and families affected by ASMD. The company stated that Xenpozyme's approval is the result of extensive research and development efforts. The drug had previously received "Breakthrough Therapy" designation from the FDA, which is intended to expedite the development and review of drugs for serious conditions.

Prior to the U.S. approval, Xenpozyme received European Commission approval in June 2022 and was approved in Japan in March 2022 under the country's SAKIGAKE designation. The FDA approval is based on positive data from the ASCEND and ASCEND-Peds clinical trials. These studies demonstrated that Xenpozyme improved lung function (measured by diffusing capacity of the lung for carbon monoxide, or DLco), reduced spleen and liver volumes, and improved platelet counts in both adult and pediatric patients, with a demonstrated safety profile.