Sanofi and its subsidiary Genzyme have receive dBreakthrough Therapy designation from the US Food and Drug Administration (FDA) for olipudase alfa, an enzyme-replacement therapy for treating nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease Type B, as opposed to type A which is characterized by neurological involvement. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase (ASM), which results in toxic accumulation of sphingomyelin. There are currently no approved treatment options for patients with Niemann-Pick Type B.

Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. The criteria for granting Breakthrough Therapy designation are preliminary clinical evidence of substantial improvement on a clinically significant endpoint over available therapies. The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Genzyme and the FDA on the olipudase alfa development program.

Olipudase alfa is being developed by Genzyme to potentially address the fundamental defect underlying the disease. Supplementing the defective or deficient native enzyme with olipudase alfa allows for the breakdown of sphingomyelin, whose accumulation is responsible for the clinical manifestation of ASMD.

The company has started enrollment of a Phase I/II pediatric study and is preparing for enrollment of a Phase II/III adult study in the second half of 2015.

Source: Sanofi