Emmaus Life Sciences announced that the Food and Drug Administration (FDA)’s Oncologic Drugs Advisory Committee voted 10–3 that the overall benefit-risk profile of Endari (L-glutamine) for the treatment of sickle cell disease was favorable.
If approved, Endari will be the first treatment for pediatric patients with sickle cell disease as well as the first treatment for adults in nearly two decades. Endari has previously received Orphan Drug and Fast Track designations from the FDA.
An efficacy study of L-glutamine (Study 09-01) showed that the treatment resulted in a lower frequency of sickle cell painful crises, longer time to first and second crisis, fewer ACS (acute chest syndrome), fewer hospitalizations, fewer days hospitalized, and fewer transfusions compared to placebo. Overall, L-glutamine was well tolerated with a safety profile similar to placebo.
The FDA takes the Committee’s advice into consideration but is not bound to its recommendation when reviewing the New Drug Application. The Agency has set a PDUFA action date for July 7, 2017.
For more information visit EmmausLifeSciences.com.