Ultomiris is said to be the first and only long-acting C5 inhibitor administered every eight weeks and it works by inhibiting the C5 protein in the terminal complement cascade.
Atypical HUS, also called as complement-mediated TMA, is a severe and chronic ultra-rare disease, which can cause progressive damage to vital organs such as kidneys. It will lead to kidney failure and premature death.
The terminal complement cascade, a part of the body’s immune system, will result in severe ultra-rare disorders such as aHUS, paroxysmal nocturnal hemoglobinuria (PNH) and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG) when activated in an uncontrolled manner.
Alexion has submitted the sBLA based on results of the phase 3 study of Ultomiris, which achieved its primary objective in complement inhibitor-naïve patients with aHUS.
According to the company, the 53.6% of patients showed complete TMA response in the initial 26-week treatment period.
The global, multicentre, single arm and phase 3 study assessed he safety and efficacy of Ultomiris dministered by intravenous infusion in 56 adults who had not been treated with a complement inhibitor before.
The trial comprised of up to seven-day screening period, a 26-week initial evaluation period and an extension period of up to two years, which is still ongoing.
Alexion research and development head and executive vice president Dr John Orloff said: “This acceptance is an important step in our efforts to deliver a potential new standard of care to people living with this devastating disease.
“We look forward to working closely with the FDA to facilitate a rapid review of this application.”
The regulator has set a target action date of 19 October 2019 under the Prescription Drug User Fee Act (PDUFA).
Alexion has already developed and commercialised two approved complement inhibitors for the treatment of patients with PNH, as well as he first and only approved complement inhibitor to treat aHUS and AchR- antibody-positive MG.