New Haven-born Alexion Pharmaceuticals has won approval from the Food and Drug Administration to market its new drug Ultomiris to children with a rare blood disorder.
Alexion, which announced the FDA’s decision this week, said it now has the first and only medicine approved in the U.S. for children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH).
The FDA approved Ultomiris for use in adults with PNH in 2018.
Ultomiris works by inhibiting a protein in the complement system, a part of the immune system that over-responds in PNH patients, leading the body to attack its own red blood cells. The drug is Alexion’s follow-up to its blockbuster drug Soliris.
Dr. John Orloff, Alexion’s executive vice president and head of research and development, said in a statement that PNH can have a profound impact on a child’s development and quality of life. Many need to miss school for infusions, blood transfusions and medical appointments.
“With its established safety and efficacy profile, Ultomiris has the potential to transform the lives of children and adolescents suffering from this devastating rare disease,” he said.
The FDA based its decision on interim results from a Phase 3 study showing Ultomiris was effective in completely inhibiting the complement system protein C5 through 26 weeks in children and adolescents up to 18 years old.
The Boston-based company, which has a large New Haven research presence, is being acquired by pharma giant AstraZeneca in a $39 billion deal expected to close in the third quarter of this year.
Contact Natalie Missakian at news@newhavenbiz.com
