The blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), is a life-threatening form of hemolytic anemia characterized by destruction of red blood cells by the body's complement system, a component of the immune system.
A priority review designation is given to products that if approved would be a significant improvement over existing therapies. The FDA has also accepted a biologics license application for Soliris.
The European Medicines Evaluation Agency (EMEA) has already accepted the Soliris application for review under the accelerated assessment procedure. Review under this procedure is provided for medicinal products of major therapeutic interest and shortens the timeframe for review by that agency.
“There currently is no therapy specifically available for the treatment of PNH, and we believe that Soliris, if approved, would represent an important option in improving the lives of patients with the disease,” said Leonard Bell, CEO of Alexion.
The biologics license application submission is based on data from a phase III trial which met all pre-specified primary and secondary endpoints with statistical significance.