European Regulators Ready to Review Emmaus’s Sickle Cell Disease Therapy
The signal was the agency’s validation of the company’s marketing authorization application (MAA) for Xyndari.
Xyndari increases the amount of free glutamine in the blood, which sickle cells use to generate anti-oxidant molecules. The molecules help neutralize oxidative stress in sickle-shaped red blood cells, allowing them to regain a healthy shape and function.
“Sickle cell disease patients in the European Union have not had access to new therapies for almost 20 years,” Yutaka Niihara, Emmaus’ chairman and chief executive, said in a press release. “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 the results of a 48-week Phase 3 clinical trial (NCT01179217) in the U.S. that ended in 2014. It covered 230 SCD patients from 5 to 58 years old who had two or more painful crises within a year of enrollment.
Researchers compared Xyndari and a placebo’s ability to decrease sickle cell crises, hospitalizations, and emergency room visits.
Xyndari led to a statistically significant 25 percent reduction in median frequency of SCD crises and a 33 percent median decrease in hospitalization rates, results showed. Improvements were reported in both children and adults. The treatment was safe and well-tolerated.
The EMA will review Xyndari under a centralized licensing procedure for all 28 member states of the European Union, Norway and Iceland. Xyndari has also received orphan drug status and an approved pediatric investigation plan (PIP) from the European regulatory agency.
While the oral L-glutamine isn’t approved in other countries, the treatment is available through an early access program (EAP) administered by myTomorrows. The EAP gives physicians in the E.U., Turkey, the Middle East and South America an opportunity to prescribe oral L-glutamine before its approval to patients who failed to respond to all other treatments.