Emmaus Seeks Approval of Endari in United Arab Emirates

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by Marisa Wexler MS |

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Endari | Sickle Cell Disease News | Treatments | Illustration of medical professional with patient

Emmaus Life Sciences is seeking the approval of Endari (L-glutamine), an oral treatment for sickle cell disease (SCD), in the the United Arab Emirates (UAE).

“We look forward to working with the U.A.E Ministry of Health to bring this important medication to our patient population in the Emirates,” George Sekulich, senior vice president of global commercialization at Emmaus, said in a press release.

Review of the company’s application is expected to take 10 to 12 months. While the application is being reviewed, patients in the UAE can be prescribed Endari on a named patient, or early access, basis.

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About 600 people with SCD live in the UAE, and an estimated 225,000 in the broader Middle East North Africa (MENA) region, according to Emmaus, which submitted applications seeking the approval of Endari in Kuwait and Saudi Arabia earlier this year.

“Endari continues be available on a named patient basis throughout the MENA region,” Sekulich said.

Yutaka Niihara, MD, chairman and CEO of Emmaus, said: “This U.A.E application submission is the first of several full marketing applications we expect to file in Gulf Cooperation Council states for Endari, to treat the numerous sickle cell disease patients in the MENA region.”

SCD is caused by genetic mutations that result in the production of an abnormal form of hemoglobin, the protein used by red blood cells to carry oxygen through the bloodstream.

The mutated version of hemoglobin causes red blood cells to become deformed and take on a sickle-like shape. It also makes them more vulnerable to a type of cellular damage called oxidative stress, which is an imbalance between the production of free radicals and the ability of the body to counteract their harmful effects, causing a buildup of oxidant molecules.

Endari works by increasing the levels of glutamine in the body. Glutamine is an amino acid, or a protein building block, that red blood cells can take up and use to build antioxidant molecules that help fight against oxidative stress.

The treatment was approved in the U.S. in 2017 for SCD in individuals 5 and older, and was recently made more available to patients in Texas who are insured through Medicaid.