placebo in the frequency of pain crises, frequency of hospitalization and duration of hospitalization,” said lead researcher Dr. Yutaka Niihara. He is chairman and CEO of Emmaus Life Sciences, the maker of Endari.
Doctors now have two medications to treat sickle cell disease, an inherited disorder that causes the body to produce abnormal red blood cells, said Dr. Caterina Minniti, director of the Sickle Cell Center for Adults at Montefiore Medical Center in New York City.
Minniti said Endari likely will be used alongside hydroxyurea, which for decades has been the only medicine available to treat sickle cell disease.
“The data shows an improvement in symptoms as far as pain, but the improvement is modest,” Minniti said of the Endari trial. “In that sense, it’s not revolutionary. It’s additive.”
Red blood cells affected by sickle cell disease are rigid and sticky, bent into shapes that resemble sickles or crescent moons. They break apart easily and die, causing anemia, according to the Mayo Clinic.
Acute pain crises are a major complication of sickle cell disease that often land patients in the hospital. The U.S. National Institutes of Health says that these events occur when sickle cells clump together in