Emmaus has announced that the company’s Marketing Authorisation 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 III randomised, double-blind, placebo-controlled, multi-centre 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 hospitalisations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days). 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.
Xyndari for the treatment of sickle cell disease will be reviewed by the EMA under the centralised licensing procedure for all 28 member states of the European Union, Norway and Iceland. Xyndari has also received orphan designation and an approved paediatric investigation plan (PIP) from the EMA.