Alexion Pharmaceuticals has won FDA approval to market an improved version of its newest drug, a move the company says will allow patients with two rare diseases to spend less time receiving treatment.
The Food and Drug Administration approved a 100 mg/mL formulation of Ultomiris — the company’s successor to its flagship drug, Soliris — for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
PNH is a rare blood disorder marked by the destruction of red blood cells, while aHUS causes blood clots and damage to blood vessel walls that can lead to organ failure and usually strikes the kidneys. The drug works by inhibiting an immune system protein known as C5 that plays a role in both diseases.
The original 10 mg./mL version of Ultomiris garnered FDA approval in December 2018.
Alexion said Monday the new version is as safe and effective as the original but improves the average annual intravenous infusion time by roughly 60 percent.
Most patients will spend six hours or less a year receiving treatment, “giving them more time to do what they enjoy,” said John Orloff, MD, who heads research and development at Alexion.
The company said it plans to make the new drug available within a few days and will remove the previous version from the market in mid-2021.
Alexion is also seeking regulatory approval to market the drug in Japan and the European Union. A decision on the latter is expected next month.
The New Haven-born company, which has a large research presence here, is also working on another version of the drug that can be administered by patients themselves by injecting it under the skin. Alexion expects to seek regulatory approval for that formulation by the end of next year.
Contact Natalie Missakian at news@newhavenbiz.com
