Genzyme announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for olipudase alfa, an enzyme replacement therapy being studied for the treatment of patients with non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B.

There are currently no approved treatment for patients with Niemann-Pick disease type B. It is thought that supplementing with olipudase alfa allows for the breakdown of sphingomyelin, whose accumulation is responsible for the clinical manifestation of ASMD.

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The Breakthrough Therapy designation was based on data from a Phase 1b study (n=5) in adults with non-neuronopathic ASMD. Study data on repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa support its further development.

Genzyme has initiated enrollment for a Phase 1/2 pediatric study and is preparing for a Phase 2/3 adult study in the second half of 2015.

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