"There is currently no approved treatment for ASMD and so today's announcement of the first patient in the UK receiving olipudase alfa in this trial is a very welcome step," said Toni Mathieson, Chief Executive at Niemann-Pick UK (NPUK). "We look forward to seeing the trial progress and to continuing to work closely with physicians, patients and companies, such as Sanofi Genzyme, who are committed to developing effective treatments for rare diseases."
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.
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Stefanie Holman Chris Gardner
Sanofi Genzyme UK & Ireland Consilium Communications
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SOURCE Sanofi Genzyme