Posted by Michael Wonder on 28 Jun 2022
Xenpozyme (olipudase alfa) approved by European Commission as first and only treatment for ASMD
28 June 2022 - The European Commission has approved Xenpozyme (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in paediatric 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) and reduction of spleen and liver volumes, with a well-tolerated safety profile.
