Alexion Pharmaceuticals Inc. won federal approval last week for its successor compound to Soliris, the blockbuster blood-disorder drug that first put the New Haven-born company on the map as a bioscience powerhouse more than a decade ago.
In a decision two months ahead of schedule, the U.S. Food & Drug Administration approved Ultomiris (formerly ALXN1210) to treat the debilitating, ultra-rare blood disease paroxysmal nocturnal hemoglobinuria (PNH). The FDA had set a Feb. 18 deadline for the ruling.
“We believe Ultomiris has the potential to become the new standard of care for patients with PNH,” said John Orloff, Alexion’s executive vice president and head of research and development.
PNH is a life-threatening disorder in which a patient’s immune system prematurely destroys red blood cells.
Soliris, which had previously been the only FDA-approved drug for the condition, generated $3.13 billion for Alexion last year, nearly 89 percent of the company’s total revenue, according to Reuters.
Clinical trials found Ultomiris equally effective in treating the disease, but a key improvement is its dosing schedule — every eight weeks instead of every two with Soliris.
“With Ultomiris, patients no longer have to plan their lives around biweekly infusions and can look forward to just six or seven infusions a year,”said Neil Horikoshi, CEO of the Aplastic Anemia and Myelodysplastic Syndrome (AAMDS) International Foundation.
Both drugs work by inhibiting c5, an immune system protein that is overactivated in PNH patients.
Alexion moved its New Haven headquarters to Boston over the summer but continues to have a significant research presence in the Elm City.
Contact Natalie Missakian at news@newhavenbiz.com
