Endari Approved to Treat Sickle Cell in Adults, Children in Qatar

Raising access to SCD treatment in 'large and underserved' areas of world

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Qatar Ministry of Public Health has approved Endari (L-glutamine) to treat adults and children, starting at age 5, with sickle cell disease (SCD), the therapy’s developer, Emmaus Life Sciences, announced.

“We are grateful that Qatari officials made access to Endari easier for the sickle cell patients in the country,” Yutaka Niihara, MD, chairman and CEO of Emmaus, said in a press release.

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“The approval from the Ministry of Public Health is another validation of the safety and efficacy of Endari. We look forward to working with our distribution partner to make Endari available to the sickle cell disease patients in the emirate,” added George Sekulich, senior vice president of global commercialization at Emmaus.

Emmaus also announced its first receipt a major purchase order from its distributor in the Kingdom of Saudi Arabia. Endari is currently available on an early access basis in Saudi Arabia; an application seeking its approval there is under review.

The therapy is also up for approval in Kuwait, and it was approved in the United Arab Emirates (UAE) earlier this year.

“We will continue … in our mission to afford increased availability of Endari in the Middle East and North Africa region with its large and underserved sickle cell disease patient population,” Niihara said.

In the U.S., Endari has been approved since 2017 as a treatment for SCD patients ages 5 and older. At the time, it was the first SCD treatment to be approved in the U.S. for children, and the first new therapy to be approved for adults with SCD in nearly two decades. Emmaus recently launched a full-service telehealth platform to help U.S. patients access Endari.

SCD is caused by mutations in a gene that provides instructions for making a component of hemoglobin — the protein that red blood cells use to ferry oxygen through the bloodstream. Abnormal hemoglobin molecules clump in red blood cells, deforming them into a “sickle” shape and rendering them more vulnerable to oxidative stress, a type of cell damage driven by highly reactive oxygen-containing molecules.

Endari, an oral therapy, is designed to increase the levels of the amino acid L-glutamine, a building block of proteins. Red blood cells can take up L-glutamine to make antioxidant molecules that reduce the effects of oxidative stress, ultimately improving the cells’ health and functionality.