13 March 2018 - - US-based therapeutics developer Emmaus Life Sciences's marketing authorization application for Xyndari has been fully validated and is now under assessment by the European Medicines Agency (EMA) for the treatment of sickle cell disease, the company said.
The MAA is supported by data from the company's phase three randomised, double-blind, placebo-controlled, multi-center clinical trial of 230 patients ages five 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, fewer hospitalizations for sickle cell pain, and fewer days in the hospital.
Study patients on Xyndari also had fewer occurrences of acute chest syndrome, a life-threatening complication of sickle cell disease (8.6% vs. 23.1%).
A sickle cell crisis was defined as a visit to an emergency room/medical facility for sickle cell disease-related pain treated with a parenterally administered narcotic or ketorolac; the occurrence of chest syndrome, priapism, and splenic sequestration were also considered sickle cell crises.
The most common adverse reactions (incidence >10%) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain and chest pain.
Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older.
Emmaus is engaged in the discovery, development, and commercialization of innovative treatments and therapies for rare diseases. The company's research on sickle cell disease was initiated by Yutaka Niihara, MD, MPH, chairman and CEO of Emmaus, at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center.