Genzyme, a Sanofi company, was granted Breakthrough Therapy designation for olipudase alfa from the FDA. This enzyme replacement therapy is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B, a serious and life-threatening disorder resulting in toxic accumulation of sphingomyelin. There are currently no approved treatments for this disease.

Breakthrough designation is intended to expedite the development and review of investigational drugs that target serious or life-threatening conditions, and is supported by data from a Phase 1b study of olipudase alfa on the repeat-dose safety, pharmacodynamics, and exploratory efficacy.

The company began enrollment of a Phase 1/2 pediatric study and will begin enrollment of a Phase II/III adult study in 1H15.