FDA Approves Novel ASMD Treatment from Sanofi

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Sanofi’s Xenpozyme (olipudase alfa-rpcp) is now the only approved treatment for acid sphingomyelinase deficiency.

Sanofi announced on Aug. 31, 2022 that FDA approved its drug, Xenpozyme (olipudase alfa-rpcp), for treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. According to a company press release, olipudase alfa-rpcp is the first therapy indicated specifically for the treatment of ASMD and the only approved treatment for the disease.

ASMD, which represents a spectrum of disease, is classified into three categories: type A, type B, and type A/B. Type A and type B sit on opposite ends of the spectrum, whereas A/B is an intermediate form. The condition can result in an enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other symptoms. According to the release, fewer than 120 patients are diagnosed with ASMD in the United States; approximately 66% of these patients are pediatric.

Xenpozyme is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy that is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme allowing for the breakdown of the lipid sphingomyelin. In individuals with ASMD, the deficiency in the ASM enzyme leads to sphingomyelin accumulation in various tissues. The drug is administered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase.

Prior to its approval, Xenpozyme received breakthrough therapy and priority review designations. According to the release, FDA based its approval on results from two clinical trials (ASCEND and ASCEND-Ped), which demonstrated clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide) and platelet count as well as reduced liver and spleen volumes. Japan and the European Union also approved the treatment in March and June of 2022, respectively.


“Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families,” said Bill Sibold, vice-president and head of Specialty Care, Sanofi, in the press release. “This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and our unwavering commitment to this historically overlooked community.”

Source: Sanofi