FDA approval granted to Emmaus for sickle cell therapy

June 9, 2017  Source: fiercebiotech 152

Emmaus Medical recently received FDA (The U.S. Food and Drug Administration) approval for sickle cell disease therapy Endari (L-gluitamine), ending two decades of drought for treatment of the disease and giving an alternative to the only other drug on the market, hydroxyurea.

Sickle cell disease is a blood disorder where red blood cells are abnormally form being sickle shaped and not round, resulting in poor blood flow and decreased oxygen release.

The trail was held for 48 weeks with participants ranging from 5 to 58 years old. All participants had experienced at least 2 pain crises in the 12 months prior to enrollment for the trail. Patients were randomly either treated with Endari and others placebo.
Endari will be the first treatment viable for both children (aged 5 and older) and adults and studies have shown that the treatment resulted in less frequent sickle cell pain crises as well as prolonged periods between crises. Results also indicated a decrease in hospitalization along with fewer days in hospital. One of the factors that most likely sealed the deal for Emmaus was the fact that acute chest syndrome (a vaso-occlusive crisis of the pulmonary vasculature and one of the more serious side effects of the disease) was seen to have a decline of 58%.
Some side effects of Endari are: coughing, nausea, constipation, headaches as well as back and chest pain.

Endari is a powder formulation to be consumed orally and is currently sold by Emmaus Medical as a nutritional product to assist with short bowl disease and is the active ingredient, L-Glutamine, is available over the counter. Other drugs from Pfizer, Novartis and GlycoMimetics are also  in development with Glycomimetics' rivipansel currently in a Phase iii trial lead by Pfizer.

By Ashley

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