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# FDA Approves New Sickle Cell Therapy
- URL: https://www.fdaweb.com/fda-approves-new-sickle-cell-therapy/
- Published: 2017-07-10T12:00:00.000Z
- Updated: 2026-09-14T22:33:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139080

FDA has approved an Emmaus Medical NDA for Endari (L-glutamine oral powder) for patients five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. “Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” CDER Oncology Center of Excellence director **Richard Pazdur** is quoted in a [release](https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm566084.htm?ref=fdaweb.com) as saying. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

  
Approval was based on data from a randomized trial in patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the past 12 months. Patients were assigned randomly to Endari or placebo, and the treatment effect was evaluated over 48 weeks. “Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days),” the release says. “Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6% vs. 23.1%).”