Medicaid to Cover Endari for Sickle Cell Disease in the U.S.

Emmaus Life Sciences has reached an agreement with the Centers for Medicare and Medicaid Services that will allow coverage of Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease (SCD) in adults and children 5 years and older.

“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,” Yutaka Niihara, MD, MPH, chairman and CEO of Emmaus, said in a press release. “Having the Medicaid Drug Rebate Program in place will make Endari more accessible to patients who need it most.”

Endari was approved in the United States in July 2017. It was the first treatment approved by the U.S. Food and Drug Administration (FDA) for pediatric patients 5 and older with SCD, and the first new treatment for the condition approved in more than 20 years.

Endari increases the amount of an amino acid called “glutamine” that circulates in the blood to help generate anti-oxidant molecules. Because SCD patients are particularly susceptible to oxidation damage, the antioxidants produced by glutamine degradation help neutralize the oxidative stress in sickle red blood cells, enabling them to return to their normal shape so they can continue carrying oxygen to tissues throughout the body.

The approval of Endari was based on safety and effectiveness data from a 48-week Phase 3 clinical trial (NCT01179217) involving people with SCD from the ages of 5 to 58 who had two or more painful crises in the previous 12 months before enrollment.

Researchers randomly assigned 230 participants to receive Endari or a placebo. Patients treated with Endari experienced fewer crisis episodes, fewer hospitalizations for SCD-associated pain and fewer days in the hospital. Patients who received Endari also had fewer occurrences of acute chest syndrome, a life-threatening complication of SCD.

Earlier this month, the FDA made Endari available by prescription to most SCD patients in the U.S. Emmaus is working with payers to ensure coverage and access for the SCD community. At this point, Endari is available by prescription through U.S. Bioservices, a national speciality pharmacy.