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Emmaus Life Sciences Receives Validation From European Medicines Agency on Marketing Authorization Application for Sickle Cell Disease Treatment Xyndari (oral glutamine)
Emmaus Life Sciences, Inc. announced today that the company’s Marketing Authorization Application (MAA) for Xyndari has been fully validated and is now under assessment by the European Medicines Agency (EMA) for the treatment of sickle cell disease.
“Sickle cell disease patients in the European Union have not had access to new therapies for almost 20 years,” said Yutaka Niihara, MD, MPH, CEO and chairman of Emmaus. “The submission of this application reflects our continued commitment to provide treatment options for this painful and life-threatening disease to as many patients as possible.”
The MAA is supported by data from the company’s phase 3 randomized, double-blind, placebo-controlled, multi-center clinical trial of 230 patients ages 5 to 58 years old with sickle cell disease who had two or more painful crises within 12 months prior to enrollment. Patients who were treated with Xyndari over a 48-week period experienced fewer crisis episodes compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.