The European Commission (EC) has approved Sanofi’s (Euronext: SAN) Xenpozyme (olipudase alfa) as the first and only enzyme replacement therapy to treat 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.
This approval is based on positive data from the ASCEND and ASCEND-Peds trials, in which Xenpozyme showed substantial and clinically-relevant improvement in lung function and reduction of spleen and liver volumes, with a well-tolerated safety profile.
"The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease"ASMD is an extremely rare, progressive genetic disease with significant morbidity and mortality, especially among infants and children, as many pediatric patients will not survive to adulthood.
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze