EU approves Xenpozyme for Niemann-Pick disease.- Sanofi
The European Commission has approved Xenpozyme (olipudase alfa), from Sanofi, as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B.
The approval is based on positive data from the ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed substantial and clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and reduction of spleen and liver volumes, with a well-tolerated safety profile.
Given the urgent unmet medical needs of the ASMD community, the European Medicines Agency (EMA) granted Xenpozyme PRIority MEdicines (PRIME) designation. Xenpozyme has also received special breakthrough designations from several other regulatory agencies around the world. Historically known as Niemann-Pick disease types A, A/B, and B, ASMD is a rare, progressive, and potentially life-threatening genetic disease.
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