The FDA has granted Orphan Drug status to Alexion Pharmaceuticals, Inc.’s humanized monoclonal antibody C5 Complement Inhibitor 5G1.1 for the treatment of patients with dermatomyositis.
Alexion is currently enrolling patients in a Phase Ib pilot clinical trial that is designed to gather clinical data regarding the safety profile and biological and clinical effects of 5G1.1 in this patient population.
The Orphan Drug designation provides Alexion with market exclusivity for seven years from the drug’s approval date.
“Dermatomyositis is a rare and severe inflammatory disorder, the rash and debilitating weakness of which we believe may be alleviated by 5G1.1,” said Leonard Bell, M.D., President and CEO of Alexion.
“We are enthused to work in collaboration with the FDA to as rapidly as possible attempt to provide an innovative therapy for this small, but underserved patient population.
We are continuing to aggressively pursue 5G1.1 in this and other potential orphan indications with the objective of providing relief to multiple underserved and previously unrecognised patient groups.”
Alexion is engaged in the discovery and development of therapeutic products aimed at treating patients with a wide array of severe disease states, including cardiovascular and autoimmune disorders, inflammation and cancer.
Alexion’s two lead product candidates are currently in eight clinical development programs.
5G1.1-SC, in collaboration with Procter & Gamble, is in a Phase IIb cardiopulmonary bypass efficacy trial and in two Phase II myocardial infarction efficacy trials.