Rare Daily Staff

The U.S. Food and Drug Administration approved Alexion Pharmaceuticals’ a long-acting C5 complement inhibitor for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare blood disorder.

PNH is characterized by destruction of the red blood cells by the part of the immune system known as the complement system. PNH can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.

Ultomiris works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like PNH, atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor antibody-positive myasthenia gravis. It is the first and only long-acting C5 inhibitor administered every eight weeks

Regulatory authorities in the European Union and Japan have accepted and are reviewing applications for the approval of Ultomiris as a treatment for adults with PNH.

In phase 3 clinical studies in complement inhibitor-naïve patients with PNH, and patients with PNH who had been stable on Soliris, intravenous treatment with Ultomiris every eight weeks demonstrated non-inferiority to intravenous treatment with Soliris every two weeks on all 11 endpoints.

Ultomiris is also currently being evaluated in a phase 3 clinical study in complement inhibitor-naïve patients with aHUS, administered intravenously every eight weeks. In addition, Alexion plans to initiate a phase 3 clinical study of Ultomiris delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS. Alexion is also planning to initiate the development of Ultomiris, intravenously administered every eight weeks, as a potential treatment for patients with generalized myasthenia gravis.

“Immediate and complete C5 inhibition with Ultomiris, sustained for eight weeks, can provide meaningful benefits for patients and their families,” said John Orloff, executive vice president and head of research and Development at Alexion. “Based on the totality of our compelling data from the largest Phase 3 program ever conducted in PNH, we believe Ultomiris has the potential to become the new standard of care for patients with PNH.”

December 21, 2018
Photo: John Orloff, executive vice president and head of research and Development at Alexion