Therapy Areas: Central Nervous System
Genzyme's Xenpozyme receives US FDA approval for treatment of Acid Sphingomyelinase Deficiency
1 September 2022 -

United States-based Genzyme announced on Wednesday, that the United States Food and Drug Administration has approved the company's Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD).

The product is the first approved medication intended for the treatment of symptoms that are not related to the central nervous system in patients with ASMD. It is an enzyme replacement therapy that helps decrease sphingomyelin accumulation in the liver, spleen, and lung.

The efficacy of the product for the treatment of ASMD was showcased in a randomised, double-blind, placebo-controlled study of 31 patients randomised to take the product or placebo. The company stated that treatment with Xenpozyme improved lung function and decreased liver and spleen size.



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