International Niemann–Pick Disease Alliance

Genzyme trial for NP-B – first paediatric patient dosed with investigational ERT

Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients:

First patient dosed with investigational enzyme replacement therapy designed to treat the nonneurological manifestations of ASMD which characterize Niemann-Pick disease type B.

Monday, June 15, 2015 8:00 am EDT: read the full article here.

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Genzyme, a Sanofi company, announced today that the first pediatric patient has begun treatment in a Phase 1/2 clinical trial focused on evaluating the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of nonneurological 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) resulting in toxic accumulation of sphingomyelin. There are currently no approved treatment options for patients with Niemann-Pick disease type B.

The Phase 1/2 trial is a multi-national, multi-center, open label, ascending dose trial to evaluate the safety, tolerability and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in paediatric patients with ASMD. Twelve paediatric patients will be enrolled into 3 age cohorts: an adolescent cohort (12 to <18 years of age); a child cohort (6 to <12 years of age); and an infant/early child cohort (birth to <6 years of age). The primary objective of the Phase1/2 trial is to assess the safety and tolerability of olipudase alfa. Upon completion of the 52-week trial, patients will have the option to enroll into an extension study. Genzyme is preparing for enrollment to begin in a Phase 2/3 trial involving adult patients with ASMD in the second half of 2015. For more information please visit

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa. Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. The criteria for granting Breakthrough Therapy designation are preliminary clinical evidence of substantial improvement on a clinically significant endpoint over available therapies. The Breakthrough Therapy 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.

“With start of clinical studies both in pediatric and adult patients just a few months apart, we are confirming our commitment to the Niemann Pick disease patient community to advance the development of this novel therapy as quickly as possible and for both patient populations,” said Head of the Sanofi Genzyme R&D Center, Jim Burns, Ph.D. “We are very grateful to the patients for their engagement and their support in advancing this exciting program.”

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