Alexion Pharmaceuticals Inc. is a step closer to gaining approval to market the latest version of its new drug, Utomiris, to patients in Europe.
The rare-disease drugmaker said Monday that an advisory committee for the European Medicines Agency, which oversees pharmaceuticals in the European Union, has issued a positive opinion on the drug.
If approved, the new 100mg/mL formulation will cut average annual infusion time for patients by more than half, the company said.
Alexion, which has a large research presence in New Haven, is seeking permission to market the drug to patients with the rare diseases paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
A final decision is expected in November.
PNH is an ultra-rare blood disorder characterized by the destruction of red blood cells, while aHUS causes progressive damage to the kidneys and other vital organs, due to blood clots and damage to the walls of blood vessels.
“The advanced formulation of Ultomiris marks continued progress in Alexion’s ongoing efforts to innovate for patients, and we expect it will represent yet another step in refining the treatment schedule for patients,” John Orloff, MD, Alexion’s research and development head, said in a statement. “Importantly, it would mean that most patients would spend only six hours or less a year receiving treatment, allowing them to focus on other aspects of their lives.”
Ultomiris is Alexion’s replacement for its blockbuster drug Soliris.
Contact Natalie Missakian at news@newhavenbiz.com
