Type of information: Granting of a Market Authorisation in the US
Product name: Endari®
Compound: Levoglutamide (L-glutamine)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Company: Emmaus Life Sciences (USA)
Disease: sickle cell disease
- • On July 7, 2017, the FDA approved Endari® (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. Endari® is the first FDA-approved treatment for pediatric patients with sickle cell disease, and the first new treatment in nearly 20 years for adult patients.
- The safety and efficacy of Endari® were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari® or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari® experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days). Patients who received Endari® also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).
- Common side effects of Endari® include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.
- • On April 18, 2017, Emmaus Life Sciences announced that the Oncologic Drug Advisory Committee of the FDA has set a date of May 24, 2017 to review the New Drug Application (NDA) for orally-administered pharmaceutical grade L-glutamine product (Endari™), for the treatment of sickle cell disease.
- • On November 30, 2016, the FDA has set a PDUFA date of July 7, 2017 for a decision on the New Drug Application for L-glutamine product for the treatment for sickle cell disease.
- • On November 8, 2016, Emmaus Life Sciences announced that the FDA has accepted for review the New Drug Application for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
- • On September 8, 2016, Emmaus Life Sciences announced it has submitted a New Drug Application (NDA) to the FDA requesting U.S. marketing approval for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
- • On July 17, 2012, Emmaus Medical, subsidiary of Emmaus Life Sciences, has announced that the European Commission (EC) has granted Orphan Medicinal Product designation for levoglutamide (L-glutamine) for the treatment of sickle cell disease. The EC designation follows the recommendation of the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products announced in May.
Emmaus said it currently is conducting a clinical trial that is in Phase III to study L-glutamine as a treatment for sickle cell disease. Research is being conducted at over 30 clinical trial sites around the United States with over 175 people presently enrolled. The company anticipates total enrollment of more than 200 and aims to complete the trial in 2013.
Submission of marketing authorization application USA : 2016-09-08
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2017-07-07
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2001-08-01
Orphan status UE: 2012-06-04
Pediatric exclusivit _USA:
Pediatric exclusivity UE: