French-based pharmaceutical company Sanofi's specialty care global business unit Sanofi Genzyme has dosed the first patient in the Ascend pivotal Phase II/III clinical trial of investigational therapy olipudase alfa to treat non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B).
Olipudase alfa is a recombinant human acid sphingomyelinase (rhASM), enzyme replacement therapy.
The multi-national, multi-centre, double-blinded, placebo-controlled Phase II/III Ascend trial is designed to determine the efficacy, safety, pharmacodynamics and pharmacokinetics profile of olipudase alfa.
Around 36 adult patients with ASMD will be enrolled in the trial and administered with olipudase alfa intravenously once every two weeks over a period of 52 weeks.
The trial will focus on determining the effect of olipudase alfa on spleen size, lung function and other important clinical parameters.
Villa Metabolica Mainz University Medical Centre principal investigator Eugen Mengel said: "ASMD is a rare and debilitating disease that can lead to serious medical conditions including failure of the lungs, liver or heart.
"The beginning of this pivotal trial is a critical milestone in the assessment of olipudase alfa's potential to impact the lives of patients living with ASMD."
Olipudase alfa addresses the fundamental defect underlying the disease and supplements the defective or deficient native enzyme, resulting in the breakdown of sphingomyelin, whose accumulation is responsible for the clinical manifestation of ASMD.
Image: Niemann pick cell displayed in spleen. Photo: courtesy of W.CC via Wikipedia.