The US FDA approved Emmaus Medical's L-glutamine for sickle cell anaemia, providing the first new drug for these patients in 20 years.
Emmaus's drug is an oral, pharmaceutical-grade amino acid. The mechanism of action of the drug is not understood, but it is thought to act by exerting antioxidant effects in red blood cells. In a 230-patient phase III trial of the drug, treated patients experienced fewer sickle cell crises, fewer hospital visits for pain and shorter stays in the hospital than placebo-treated patients.
Analysts expect the company to charge US$11,000–18,000 per year for the drug. The amino acid is also available for over-the-counter purchase as a diet supplement.
Around a dozen other sickle cell candidates are in development, including small-molecule anti-sickling agents and stem cell treatments (Nat. Rev. Drug Discov. 15, 593–594; 2016).
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Mullard, A. FDA approves first new sickle cell drug in 20 years. Nat Rev Drug Discov 16, 519 (2017). https://doi.org/10.1038/nrd.2017.149
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DOI: https://doi.org/10.1038/nrd.2017.149