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FDA Advisory Committee Recommends Approval of Endari™ from Emmaus Life Sciences for the Treatment of Sickle Cell Disease
Emmaus Life Sciences Inc. announced today that the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted 10 to 3 that the overall Benefit-Risk profile of EndariTM for the treatment of sickle cell disease (SCD) is favorable. The FDA has set a PDUFA target action date for July 7, 2017.
If approved, Endari would be the first FDA-approved treatment for pediatric patients with SCD, and the first new treatment in almost 20 years for adult patients. The therapy is an orally-administered pharmaceutical grade L-glutamine.
The FDA is not bound by the Committee’s recommendation, but takes its advice into consideration when reviewing New Drug Applications. Endari has received Orphan Drug designation in the U.S., Orphan Medicinal Product designation in the EU and Fast Track designation from the FDA.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.