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 inten...