Endari Oral Therapy Approved in UAE for SCD, Emmaus Announces

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by Steve Bryson, PhD |

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The United Arab Emirates (UAE) Ministry of Health has approved Endari (L-glutamine) for sickle cell disease (SCD), according to Emmaus Life Sciences, the therapy’s developer.

During the five-month review process, which was originally expected to take up to one year, the oral therapy was available by early access only.

“We are grateful to have received the Ministry of Health’s approval of Endari for the treatment of sickle cell disease patients in the U.A.E. and will move as quickly as possible to reach this population,” Yutaka Niihara, MD, chairman and CEO of Emmaus, said in a press release.

The company estimates there are up to 600 people with sickle cell disease living or working in the UAE, and about 225,000 patients across the Middle East North Africa (MENA) region who may be eligible to be treated with Endari.

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Emmaus also submitted applications in Kuwait and Saudi Arabia in 2021 seeking Endari’s approval in those countries.

“This is the first of several full marketing approvals we hope to obtain in Gulf Cooperation Council states, which represent a very important market, given that there are more than double the number of sickle cell disease patients within these areas than there are in the United States,” Niihara said.

The U.S. Food and Drug Administration approved Endari in 2017 to treat SCD patients, ages 5 and older. It was the first approved treatment for SCD children in the U.S., and the first new therapy to be approved for adults with the disease in almost 20 years.

“After the extensive review by the Ministry of Health, the approval to market Endari is an important step forward for Emmaus in the MENA region. We look forward to working with our distribution partner to make Endari available to sickle cell disease sufferers in the U.A.E.,” added George Sekulich, senior vice president of global commercialization at Emmaus.

SCD is caused by mutations in the HBB gene that provides instructions for making a component of hemoglobin — the protein in red blood cells that carries oxygen. Abnormal hemoglobin causes red blood cells to become more susceptible to oxidative stress — a form of cellular damage resulting from an imbalance in the levels of harmful oxidant molecules and the body’s antioxidant defense mechanisms. That leads to red blood cells, normally oval-shaped, taking on the sickle-like shape that gives the disease its name.

L-glutamine is an amino acid — a protein building block — that also plays a role in energy and antioxidant generation and is produced in response to infection, injury, and stress.

Endari is designed to increase the amount of L-glutamine circulating in the bloodstream, which can be taken up by sickled red blood cells to counteract the effects of oxidative stress, allowing these cells to become more flexible and improving their ability to transport oxygen.