FDA Approves First New Sickle Cell Therapy in Nearly 20 Years

The U.S. Food and Drug Administration approved L-glutamine oral powder for patients ≥5 years old with sickle cell disease (SCD). It is the first drug approved for SCD in nearly 20 years. Hydroxyurea, which was approved in 1998, is the only other FDA-approved medication for reducing the acute complications of SCD.

The approval was based on results of a randomized trial of 298 patients (age range = 5-58 years old) who had ≥2 painful crises within the 12 months prior to study enrollment. Patients were randomized to receive L-glutamine oral powder or placebo and were followed for 48 weeks. Patients treated with L-glutamine oral powder had fewer hospital visits for sickle cell crises (median = 3 visits; range not provided), compared with those receiving placebo (median = 4 visits; range not provided). They also had fewer hospitalizations for sickle cell pain (median = 2 vs. 3, respectively; range not provided), and fewer days spent in the hospital (median = 6.5 vs. 11 days, respectively; range not provided). Patients receiving L-glutamine oral powder also had fewer occurrences of acute chest syndrome (8.6%), compared with those receiving placebo (23.1%).

The most common adverse events associated with L-glutamine oral powder were constipation, nausea, headache, abdominal pain, cough, pain in the extremities, and back and chest pain.

Source: U.S. Food and Drug Administration news release, July 7, 2017.