Endari approved in Oman for adults, children with sickle cell disease
The disease affects about 3,000 people in the country
The disease affects around 3,000 people in Oman, with about 6% of the population estimated to carry genetic mutations associated with it.
“We are delighted to receive marketing authorization for Endari in Oman, where sickle cell disease is a significant public health issue that affects thousands of patients and their families,” Yutaka Niihara, MD, chairman and CEO of Emmaus, said in a company press release. “This approval marks another milestone in our mission to improve the lives of people with sickle cell disease around the world.”
SCD is caused by mutations in the beta-globin gene (HBB), which contains instructions to produce a component of hemoglobin — a protein in red blood cells responsible for transporting oxygen. The mutations result in a defective form of the protein being produced.
Faulty hemoglobin molecules then clump inside red blood cells, causing them to acquire a sickle-like shape and making them more susceptible to oxidative stress, a type of cell damage caused by highly reactive oxygen-containing molecules.
How does Endari work and where else is it approved?
Endari is designed to increase the levels of the amino acid L-glutamine, a protein building block. Red blood cells can take up this amino acid and use it to produce antioxidant molecules that can help neutralize oxidative stress, improving cells’ health and function.
Endari has been approved in the U.S. to treat SCD in people ages 5 and older since 2017. At the time, it became the first therapy approved in the country to treat children and the first to be approved for SCD in nearly 20 years. Last year, Emmaus released a full-service telehealth platform to give patients easier access to Endari.
The therapy is also approved in Israel, Bahrain, Kuwait, Qatar, and the United Arab Emirates. In Saudi Arabia, where its approval is under review, Endari is available on an early access basis. The early access program lets physicians use investigational therapies before they’re formally approved when no comparable treatments are available.
The therapy is available in France and the Netherlands on a named patient basis, wherein it can be requested by a physician directly from the manufacturer for an individual patient, or via early access.
“The Omani marketing approval for Endari is an important step forward for us in the [Gulf Cooperation Council] region, where we have been working closely with the local authorities, physicians, and patient groups to ensure access to this innovative treatment for sickle cell disease,” said George Sekulich, senior vice president of global commercialization at Emmaus.