FDA Approves First New Sickle Cell Treatment in Nearly 20 Years

The U.S. Food and Drug Administration (FDA) has approved L-glutamine oral powder to reduce severe complications associated with sickle cell disease (SCD) in patients ≥5 years old. Hydroxyurea, which was approved in 1998, is the only other FDA-approved medication for reducing the acute complications of SCD.

The decision was based on data from a phase III trial of 298 patients (age range = 5-58 years) with SCD who had two or more painful sickle cell crises in the 12 months prior to enrollment. Patients were randomized 2:1 to receive oral L-glutamine 0.3 mg/kg/day (n=151) or placebo (n=78) for 48 weeks, followed by a three-week tapering period.

During follow-up, patients in the L-glutamine arm experienced:

  • fewer hospital visits for sickle cell crises (median = 3 vs. 4)
  • fewer hospital visits for sickle cell pain (median = 2 vs. 3)
  • fewer days in the hospital (median = 6.5 vs. 11)
  • fewer cases of acute chest syndrome (8.6% vs. 23.1%)

Common adverse events included constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain, and chest pain.

Source: FDA press release, July 7, 2017.

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