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Posted by on Jun 4, 2015 in Foundation NewsLine |

NNPDF ASMD News: FDA Grants Breakthrough Therapy Designation for Genzyme’s Olipudase Alfa

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Dear NNPDF Families and Friends,
The NNPDF Central Office is pleased to share a Press Release from Genzyme, a Sanofi Company, (dtd:  Thursday, June 4th, 2015) which announces that the United States Food and Drug Administration (FDA) has granted “Breakthrough Therapy” designation to olipudase alfa. This enzyme replacement therapy (ERT) is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease type B. 
Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions which have an unmet therapy or medical treatment. 
Please visit the NNPDF Newsline page ( for access to the full press release, as well as, to learn more information about the FDA “Breakthrough Therapy” designation and the impact that this FDA designation will have on the upcoming pediatric and adult clinical trials in the United States.

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