FDA approves first treatment for acid sphingomyelinase deficiency, a rare genetic disease

FDA

31 August 2022 - Today, the US FDA approved Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with acid sphingomyelinase deficiency, a rare genetic disease that causes premature death.

Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with acid sphingomyelinase deficiency.

Read FDA press release

Michael Wonder

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Michael Wonder

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Outcome , Medicine , US , Orphan drug