The FDA announced Friday that it approved Emmaus Medical's Endari (L-glutamine) for the reduction of severe complications associated with sickle cell disease in patients aged five years and older. Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said the oral powder "is the first treatment approved for patients with sickle cell disease in almost 20 years," noting that "until now, only one other drug was approved for patients living with this serious debilitating condition."
Emmaus' marketing application for Endari was supported by data from a Phase III trial involving 230 adult and paediatricpatients with sickle cell disease who had experienced at least two painful crises within the 12 months preceding enrollment. Patients were randomised to receive Endari or placebo, and the effect of treatment was evaluated over 48 weeks.
According to the FDA, patients in the Endari group made a median three hospital visits for pain treated with a parenterally administered narcotic or ketorolac, compared to a median four visits for those on placebo. On average, Endari-treated patients were also hospitalised two times for sickle cell pain and spent 6.5 days in hospital, whereas the placebo group had a median three hospitalisations lasting a median 11 days. Moreover, patients who received Endari experienced fewer occurrences of acute chest syndrome versus placebo, with rates of 8.6 percent and 23.1 percent, respectively.
In May, an FDA advisory panel voted 10-3 that the benefits of Endari outweigh its risks. The agency noted that it had previously awarded Endari orphan drug status for sickle cell disease. The therapy also has an orphan drug designation in the EU for the same indication, and Emmaus has said it intends to submit a marketing authorisation application to the European Medicines Agency.
For related analysis, see KOL Views: Endari's clinical benefit for SCD may end up trumping its commercial value, says leading expert.
To read more Top Story articles, click here.