FDA approves olipudase alfa-rpcp for acid sphingomyelinase deficiency


Olipudase alfa-rpcp (Xenpozyme; Sanofi) is the first disease-specific, and now, only approved therapy for the treatment of acid sphingomyelinase deficiencies in pediatric and adult patients.

Sanofi announced the US Food and Drug Administration (FDA) has approved olipudase alfa-rpcp (Xenpozyme) for the 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.1 Olipudase alfa-rpcp is now the first disease-specific and FDA-approved treatment for ASMD (non-CNS manifestations).1

Olipudase alfa-rpcp, is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy intended to replace deficient or defective acid sphingomyelinase (ASM). ASM is an enzyme that permits the breakdown of the lipid sphingomyelin.

Niemann-Pick disease (types A, A/B, and B), otherwise known as ASMD, is a rare genetic disease with significant morbidity and mortality. Fewer than 120 patients are estimated to be diagnosed with ASMD in the US, and roughly two-thirds of US patients with ASMD are pediatric.

Common symptoms of ASMD present in infancy, childhood, or adulthood, and include enlarged spleen or liver, lung infections, difficulty breathing, and unusual bruising or bleeding.

In patients with ASMD, this deficiency leads to sphingomyelin accumulation in several tissues. Investigators state that olipudase alfa-rpcp does not modulate CNS manifestations of ASMD or is expected to cross the blood-brain barrier. The company notes that olipudase alfa-rpcp has not been studied in patients with ASMD type A.

“Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families,” said Bill Sibold, executive vice president and head of Specialty Care at Sanofi. “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.”

The approval was based on positive data from ASCEND and ASCEND-Peds clinical trials. Data from these trials demonstrated a clinically relevant improvement in lung function and platelet count, a reduction of spleen and liver volumes, and a demonstrated safety profile within study participants receiving olipudase alfa-rpcp.

“ASMD is an extremely rare, progressive, and potentially fatal genetic disease that impacts children and adults around the world,” said Melissa Wasserstein
MD, pediatric genetic medicine at Albert Einstein College of Medicine and the Children’s Hospital at Montefiore. “Until now, those living with ASMD have had no FDA-approved treatment to combat this devastating condition. I’m proud of the work that has been done and look forward to witnessing the impact that this treatment may have on those living with ASMD.”


1. Xenpozyme (Olipudase alfa-rpcp) approved by FDA as first disease-specific treatment for ASMD (Non-cns manifestations). Sanofi. August 31, 2022. Accessed September 1, 2022. https://www.sanofi.com/en/media-room/press-releases/2022/2022-08-31-18-30-00-2507978

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