Rare Disease Report

Medicare and Medicaid Allow Endari Coverage

JANUARY 29, 2018
Mathew Shanley
This morning, Emmaus Life Sciences announced that it has a Medicaid Drug Rebate Agreement with the Centers for Medicare and Medicaid Services, allowing coverage of Endari (L-glutamine oral powder).

The news comes one week after the company announced the availability of the therapy in the United States. In July, the U.S. Food and Drug Administration (FDA) approved Endari, making it the first regulated treatment for SCD in 20 years for use in adults, and the first ever for pediatric patients. Previously, Hydroxyurea had been used as the standard of care for treating episodes of pain.

“This treatment is considered very important by many providers and patients because of its excellent safety profile and the evidence of effectiveness from the phase III study that showed a reduction in acute chest syndrome, decreased occurrence of sickle crises and decreased frequency of hospitalizations in the Endari group compared to the placebo group,” said Yutaka Niihara, MD, MPH, Chairman and CEO of Emmaus Life Sciences in a press release. “Having the Medicaid Drug Rebate Program in place will make Endari more accessible to patients who need it most.”

At the 59th American Society of Hematology (ASH) Meeting and Exposition in Atlanta in December, Emmaus announced the availability of the treatment and presented the data that led to the approval. Rare Disease Report sat down with Dr Niihara to discuss the adverse events (AEs) experienced by those enrolled in the clinical trial.


“One of the primary adverse events in the affected the treatment group was the bloating in the abdomen, and that is a common side effect of this type of product,” he said. “But these were relatively easily relieved by things like fiber or a laxative or stool softeners.”

Other common adverse reactions experienced in clinical studies included constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain and chest pain.

SCD is a rare genetic condition in which patients have red blood cells that are hard, sticky, and C-shaped. These irregular cells clog smaller blood vessels, resulting in insufferable pain and an amplified risk for infection, acute chest syndrome and stroke.

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