Emmaus Seeks Approval of Oral Medication Endari in Kuwait
The company announced that the Kuwait Drug and Food Control administration, which is responsible for registering pharmaceutical products in the country, has accepted its request for fast-track review of Endari. A decision from the regulatory authority is expected within one year.
“We are pleased to announce the submission of our marketing authorization application to the Kuwaiti authorities,” Yutaka Niihara, MD, chairman and CEO of Emmaus, said in a press release.
The application, which comes a few months after a similar application was submitted to regulatory authorities in Saudi Arabia, “represents another important milestone in our progress and commitment to serve sickle cell disease patients in the Middle East and North Africa region,” Niihara added.
Emmaus estimates there are approximately 500 people with SCD living in Kuwait. In the broader Middle East and North Africa region, known as MENA, there are an estimated 225,000 people with the disease.
“We continue to work on building relationships with hematologists and patient advocacy groups and collaborating with clinicians to provide Endari on a named-patient basis in Kuwait and the greater MENA region,” said George Sekulich, senior vice president of global commercialization at Emmaus.
Endari, which is administered as an oral powder, is designed to help red blood cells combat oxidative stress — a type of cell damage that is increased in SCD patients and known to contribute to the dysfunction of red blood cells.
Emmaus sponsored a Phase 3 clinical trial (NCT01179217) that evaluated the treatment in 230 people with SCD, ages 5–58, who had two or more painful vaso-occlusive crises in the year prior to study enrollment. Participants were given Endari or a placebo for nearly a year.
Results from the trial showed that Endari reduced the frequency of pain episodes and hospitalizations compared with a placebo. Participants given Endari also had fewer occurrences of a serious disease complication called acute chest syndrome, which can cause breathing difficulties and lead to respiratory failure.
Endari was approved in the U.S. in 2017 to treat severe SCD complications in adults and children as young as 5. It was also approved for the same indication in Israel last year.
After a branch of Europe’s regulatory agency recommended against approving Endari, Emmaus withdrew its regulatory application for Endari in Europe in 2019. The company is now exploring other paths for clinical development in Europe.
In the meantime, the medication, to be sold under the brand name Xyndari, is currently available through early access programs in a number of European Union member states, as well as in various regions in the Middle East and South America.