FDA Approves Olipudase Alf a-rpcp for Acid Sphingomyelinase Deficiency

• Published in: Drug Topics 2022-11, Vol.166 (11), p.38-38
• Main Authors: Tran, Tiana, Chamberlin, Kevin
• Format: Article
• Language: English
• Subjects: Adults; Drug dosages; FDA approval; Genetic disorders; Pediatrics; Pharmacy; Spleen
• ISSN: 0012-6616; 1937-8157

Byweek 104, patients who were initially in the placebo group had received olipudase alfa-rpcp for 52 weeks and demonstrated the following mean changes from baseline: predicted diffusion capacity of the lungs for carbon monoxide (DLco) increased by 26.8%; spleen volume reduced by 36.5%; liver volume reduced by 29.5%; and platelet count increased by 19.5%. Reconstituted olipudase alfa-rpcp vials not used immediately and diluted solutions not to be administered immediately should be refrigerated at 2 to 8 °C (36-46 °F) for up to 24 hours, or at room temperature at 20 to 25 °C (68-77 °F) for up to 12 hours. ¦ n August 31, 2022, the FDA approved olipudase alfa-rpcp (Xenpozyme) for intravenous (IV) infusion in pediatric and adult patients with acid sphingomyelinase deficiency (ASMD),1 a rare genetic disease that causes premature death due to the accumulation of a complex lipid called sphingomyelin. Olipudase alfa-rpcp received fast track, breakthrough therapy, priority review, and orphan drug designations from the FDA.1 TianaTran,PharmD,isaPGY' pharmacy resident at UConn John Dempsey Hospital in Farmington, Connecticut Kevin Chamberlin, PharmD, FASCP, is university director of pharmacy residency programs at UConn Health in Farmington, Connecticut For references, visit drugtopics.com.