Genzyme Secures FDA Breakthrough Designation

Genzyme said last week that it has received FDA breakthrough therapy designation for olipudase alfa, an enzyme replacement therapy for patients with nonneurological manifestations of acid sphingomyelinase deficiency, also known as Niemann-Pick disease type B.

ASMD is caused by a breakdown of the enzyme acid sphingomyelinase, resulting in an accumulation of enzyme that can be life-threatening. Genzyme says olipudase alfa will aid in breaking down this toxic accumulation.

There are currently no approved treatment options for patients who have nonneuronopathic ASMD.

The designation is supported by data from a study of five adult patients. Genzyme has started enrollment in a pediatric study and expects to begin enrolling patients in another adult study later this year. — John Bechtel