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FDA Approves Oral Powder for Sickle Cell Disease

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L-glutamine is the first SCD treatment approved for pediatric patients, and the first adult treatment approved in nearly 20 years.

The US Food and Drug Administration (FDA) has approved L-glutamine oral powder (Endari) for the treatment of sickle cell disease (SCD) complications in adult and pediatric patients aged 5 years or older.

The treatment is the first approved by the FDA for pediatric SCD patients and the first new adult SCD treatment in nearly 2 decades.

The powder, produced by Emmaus Medical, Inc., was approved following a randomized, double-blind, placebo-controlled, multi-center clinical trial involving 230 sickle cell anemia or sickle beta-thalssemia patients. The patients, who had to have had at least 2 painful crisus within 12 months fo enrollment, were randomized to receive either L-glutamine or placebo for 48 weeks followed by three weeks of drug tapering.

Over the testing perioud, L-glutamine patients reported a median of 3 sickle cell crises, compated to the placebo patients' median of 4 crises. A crisis was defined as a disease-related medical visit for pain, treated with parenteral narcotic or ketorolac. These crises included chest syndrome, priapism, and splenic sequestration.

L-glutamine patients also reported fewer hospitalizations from sickle cell pain, fewer cumulative hospital days, and a lower acute chest syndrome incidence.

Patients reported common adverse effects such as constipation, nausea, headaches, abdominal pain, cough, and more at least 10% while taking L-glutamine versus placebo.

The recommended dose of L-glutamine is 10-30 grams twice per day, taken orally, mixed in with either drink or food.

A press release regarding the approval was made available.

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