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FDA approves first treatment for acid sphingomyelinase deficiency

FDA approves first treatment for acid sphingomyelinase deficiency

The U.S. Food and Drug Administration has approved Xenpozyme (olipudase alfa) for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), a rare genetic disease.

ASMD is due to having less an enzyme had a need to breakdown the complex lipid sphingomyelin, that may accumulate in the liver, spleen, lung, and brain. Xenpozyme infusions provide .

The approval, which received fast-track, breakthrough therapy, orphan drug, and priority review designations, was predicated on a randomized controlled study of 31 patients where treatment with Xenpozyme was found to boost lung function and reduce liver and spleen size. In the clinical trial, 75 percent of and 1 / 2 of experienced effects, including headaches, nausea, and vomiting, while receiving the Xenpozyme intravenous infusion.

“ASMD includes a debilitating influence on people’s lives and there’s a critical have to increase treatment plans for patients who have problems with this rare disease,” Christine Nguyen, M.D., from the FDA Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a statement. “The challenges associated with developing treatments for rare diseases are significant and unique. We believe patients who have problems with ASMD, their own families, and their physicians will welcome this long-awaited advancement.”

Approval of Xenpozyme was granted to Genzyme.

More info: FDA Approval

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Citation: FDA approves first treatment for acid sphingomyelinase deficiency (2022, September 5) retrieved 5 September 2022 from

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