Search SciPol

Brought to you by
July 10, 2017

FDA approves first new sickle cell drug in 20 years

  • Government
  • Agency
  • Industry
  • Genetics/Genomics

STAT – The FDA approved a new medicine Friday to reduce the complications associated with sickle cell disease, a rare blood disorder.

The drug, Endari, is made by privately held Emmaus Medical and is the first new treatment for sickle cell disease to secure FDA approval in almost 20 years. However, the active ingredient in Endari — L-glutamine — is an old chemical that can be purchased over the counter, which could complicate Emmaus’s ability to obtain insurance coverage.

In Emmaus’s pivotal clinical trial, treatment with Endari over 48 weeks reduced the frequency and length of hospital visits for sickle cell pain crises compared to placebo. Commonly reported side effects of the drug included constipation, nausea, and headache.

Read more at STAT.

Image Citation