FDA approves Sanofi’s new ASMD treatment

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

Sanofi announced on August 31, 2022 that the FDA had approved its drug, Xenpozyme (olipudase alfa-rpcp), for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult patients and pediatrics. 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 diseases, is classified into three categories: type A, type B and type A/B. Type A and type B are at opposite ends of the spectrum, while A/B is an intermediate form. The disease can cause 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 that allows the breakdown of lipid sphingomyelin. In people with ASMD, the deficiency of the ASM enzyme leads to accumulation of sphingomyelin 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, the FDA based its approval on the results of two clinical trials (ASCEND and ASCEND-Ped), which demonstrated clinically relevant improvement in lung function (measured by the carbon monoxide diffusing capacity of the lung). and platelet count as well as decreased liver and spleen size. Japan and the European Union also approved the treatment in March and June 2022, respectively.

“Teams at Sanofi are 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. “It 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 neglected community.

Source: Sanofi

Irene B. Bowles