Breakthrough designation for Genzyme's olipudase alfa from FDA

French pharma major Sanofi’s (Euronext: SAN) subsidiary Genzyme has been granted Breakthrough Therapy designation for its investigational drug olipudase alfa by the US Food and Drug Administration.

This enzyme replacement therapy is being investigated for the treatment of patients with non-neurological 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 disease type B.

Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. This 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.