EMA Recommends Approval of Niemann-Pick Disease Types A/B and B Therapy

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EMA has recommended the marketing authorization of Xenpozyme (olipudase alfa) in the European Union.

The European Medicines Agency (EMA) has provided its recommendation for Xenpozyme (olipudase alfa) to receive marketing authorization in the European Union (EU) for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), which is a rare and progressive genetic disease.

According to a May 20, 2022 press release, the therapy is indicated for use in children and adults of all ages who suffer from type A/B or type B ASMD—a genetic disorder that is historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B). Currently, there are no approved medicines that modify or slow the progression of the disease within the EU, meaning that patients are only able to receive palliative and supportive care to manage the symptoms.

Xenpozyme is an enzyme replacement therapy, available as a powder solution for intravenous administration every two weeks, that works by replacing the deficient or defective enzyme in patients. The replacement enzyme, which is produced by recombinant DNA technology, then lowers the fat accumulation in cells and relieves disease symptoms.


The recommendation for marketing authorization, made by EMA’s Committee for Medicinal Products for Human Use (CHMP), was based on the results from three clinical trials in patients with ASMD, which demonstrated positive outcomes of the therapy with only mild to moderate adverse events experienced. The therapy has been supported through EMA’s Priority Medicines (PRIME) scheme and CHMP reviewed the marketing authorization application under an accelerated assessment procedure.

Source: EMA