Emmaus Life Sciences said last week that the FDA approved its L-glutamine oral powder, Endari, as a therapy to lessen the complications of sickle cell disease in patients ages 5 and older.
The drug’s approval was supported by data from a Phase III trial evaluating Endari compared to a placebo in 230 adults and children with sickle cell disease. The data showed that Endari cut the frequency of sickle cell crises by one-fourth and hospitalizations by 33%, according to Emmaus.
Endari’s safety profile was comparable to placebo and the compound was well-tolerated in adults and kids.
“The approval of Endari is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children,” chairman & CEO Dr. Yutaka Niihara said in prepared remarks. “Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases. We thank the FDA for its prompt review and look forward to making treatment available to patients as early as this fourth quarter.”
The active ingredient in Endari is L-glutamine, which can be purchased over the counter. This could make it challenging for Emmaus to win insurance coverage for the newly-approved product.
Sickle cell disease is a rare disorder that is recognized by its strangely-shaped red blood cells. These cells clog a patient’s vessels, cutting off the flow of oxygen to the body’s tissues.
“A sickle cell crisis is the most common acute complication for patients and the number one cause of emergency room visits,” Dr. Wally Smith, from the Virginia Commonwealth University, said. “Endari has clinically shown to reduce sickle cell crises and hospitalizations, representing a significant medical advancement for patients with limited therapeutic options that have many side effects.”