In July, the U.S. Food and Drug Administration (FDA) approved the use of Endari (L-glutamine oral powder) for patients 5 years and older with sickle cell disease. It was the first approval for the indication in 20 years, and the first ever for children.
At the 59th American Society of Hematology (ASH) Meeting and Exposition in Atlanta this week, Emmaus announced the availability of the treatment and presented the data that led to the approval. Rare Disease Report sat down with Yutaka Niihara, M.D., MPH, Chairman and CEO of the company to discuss the clinical trials for Endari, the adverse events (AEs) experienced by those enrolled, and when the drug will be available to patients.
Niihara: Sickle Cell disease is one of the most well-described genetic diseases, and it's one of the most devastating diseases. All these years, we’ve had only hydroxyurea as the disease-modifying agent. Of course, having hydroxyurea is better than nothing, but we really needed other options. All these years we've had nothing approved for pediatric patients, so we’re hoping that from here on, there will be increased interest in discussion and there will be more involvement to treat this disease.
Our purpose was to try to reduce the complications of sickle cell disease especially the painful crisis; we also call this the vaso-occlusive crisis. In turn, we have shown and proven that Endari does decrease the frequency of painful crisis, but it also decreases the frequency of hospitalization, decreases the total number of days in the hospital ,and also reduces the occurrence of one of the most devastating complications called acute chest syndrome. One of the primary adverse events in the affected the treatment group was the bloating in the abdomen, and that is a common side effect of this type of product. But these were relatively easily relieved by things like fiber or a laxative or stool softeners. We want to move on to try to show that this is efficacious for infants, too, because our studies included children 5 years and older and we want to extend this to infants ages 6 months to 5 years of age.
Right now, we have the approval in the United States but we want to get this out to everywhere in the world where sickle cell patients are.