Xenpozyme

August 31, 2022

Xenpozyme™ (olipudase alfa-rpcp) has been approved for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients.

  • Xenpozyme, manufactured by Sanofi, is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease.
  • ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement.
  • Xenpozyme is administered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase.
  • It is expected that Xenpozyme will be available in the S. in the coming weeks. The U.S. list price, or wholesale acquisition cost, of Xenpozyme is $7,142.00 per vial.

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