FDA Advisory Committee recommends approval of Emmaus Life Sciences’s sickle cell disease drug
May 24, 2017The Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted 10 to 3 that the overall Benefit-Risk profile of Emmaus Life Sciences’s Endari for the treatment of sickle cell disease (SCD) is favorable.
The company said that the FDA has set a PDUFA target action date for July 7, 2017.
“We are encouraged by the positive outcome of today’s Advisory Committee meeting and thank the committee members and FDA for their time to discuss Endari’s potential as a treatment for patients with sickle cell disease. We also appreciate and thank the patients, caregivers and advocates for the important real-world perspective they provided during the Open Public Hearing,” said Yutaka Niihara, MD, MPH, Chairman and Chief Executive Officer of Emmaus Life Sciences.”
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 company noted. 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.
