Emmaus Life Sciences Expands Distribution of Sickle Cell Disease Therapy, Endari

Catarina Silva, MSc avatar

by Catarina Silva, MSc |

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FDA approved Endari

McKesson Plasma and Biologics LLC will distribute Endari, a therapy indicated to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. The announcement was made by the Endari’s developer, Emmaus Life Sciences, Inc..

This distribution agreement enables the SCD therapy to be on all the U.S. pharmacies that use McKesson — the industry’s oldest, largest and most experienced pharmaceutical distributor — as their primary and exclusive medicine supplier. Endari is now available in more than 30 states.

“As the demand for Endari continues to increase, we seek to build our distribution network to ensure that we reach patients in need,” Yutaka Niihara, MD, MPH, CEO and chairman of Emmaus, said in a press release. “Teaming up with a premier distributor like McKesson not only expands our reach but also lends support in establishing Endari as an important treatment option for clinicians fighting this terrible disease.”

Endari is an orally administered powdered form of the amino acid L-glutamine. It works by increasing the amount of glutamine in the blood, which in turn helps to produce anti-oxidant molecules. In SCD, oxidative stress is increased and plays a significant role in the disease mechanism of SCD‐related red blood cells’ stiffness, blood vessels blockage, and organ damage. The L-glutamine amino acid in Endari reduces oxidative stress and the disease’s acute complications by easing cell stiffness to improve tissue oxygenation.

Endari was approved by the U.S. Food and Drug Administration (FDA) last summer. It was the first treatment approved for SCD children older than 5 years, and the first new therapy for the condition authorized in more than 20 years.

FDA approval was based on the results of a randomized, placebo-controlled, multi-center, Phase 3 trial (NCT01179217) evaluating the therapeutic effects of Endari in 230 sickle cell patients (age 5 to 58 years) who had two or more disease-related pain crises within 12 months prior to enrollment.

Subjects were assigned randomly to receive Endari or placebo twice a day for 48 weeks. Results showed that Endari reduced SCD-related acute complications, such as acute chest syndrome. Participants taking Endari had significantly fewer pain crises and hospitalizations due to painful complications.

Earlier this year, Emmaus reached an agreement with the Centers for Medicare and Medicaid Services to allow coverage of Endari.

Besides the distribution agreement with McKesson, Emmaus also recently partnered with the DMS Pharmaceutical Group to distribute Endari to SCD patients in the United States military, benefiting 9.4 million U.S. Department of Defense beneficiaries.