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CHMP recommends Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD).

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Published:23rd May 2022

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Xenpozyme (olipudase alfa), recommending that this investigational enzyme replacement therapy be approved in the European Union (EU) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B.

The positive CHMP opinion is based on data from the ASCEND and ASCEND-Peds clinical trials, demonstrating that Xenpozyme showed robust and clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and reduced spleen and liver volumes, with a well-tolerated safety profile in adults and children with ASMD.

The EMA previously awarded olipudase alfa the PRIority MEdicines designation, also known as PRIME, and the application was reviewed under the EMA’s accelerated assessment, intended to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. The European Commission will review the CHMP recommendation and is expected to make a final decision in the coming months.

Condition: Niemann Pick Disease/ASMD
Type: drug

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