FDA nod for first sickle cell disease drug in 20 years

7 July 2017

The US Food and Drug Administration (FDA) on Friday approved Endari (L-glutamine oral powder) for sickle cell disease to reduce severe complications associated with the blood disorder.

Sickle cell disease affects around 100,000 Americans, who face a life expectancy of around 40 to 60 years, and developing a treatment has proved highly challenging.

The company behind Endari is US biotech company Emmaus Life Sciences, which focuses on orphan diseases.

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