CAMBRIDGE, Mass.--(BUSINESS WIRE) - Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first adult patient has enrolled and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B).
ASMD is one of a group of lysosomal storage disorders that affect cellular metabolism and are caused by genetic mutations. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase resulting in accumulation of sphingomyelin in multiple organs of the body. Common clinical manifestations include enlarged liver and spleen, liver dysfunction, infiltrative lung disease, bleeding complications, cardiovascular and bone disease, and growth delay. There are currently no approved treatment options for patients with ASMD.
ASCEND is a Phase 2/3 multi-national, multi-center, double-blinded, placebo-controlled trial to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in adult patients with ASMD, specifically NPD B. The Phase 2/3 trial will assess the effect of olipudase alfa on spleen size, lung function and other important clinical parameters. Thirty-six patients are expected to be enrolled in the study and receive olipudase alfa or a placebo. Upon completion of the 52 week primary analysis period, all patients will receive treatment in an extension period.
Read more: http://news.genzyme.com/press-release/sanofi-genzyme-begins-pivotal-phase-23-trial-olipudase-alfa-adult-patients-acid-sphing