AstraZeneca shares were up 0.4% to 10,553.6p in early morning trading on Thursday, following the firm’s announcement that its Ultomiris treatment had been approved in the US.
The treatment was developed for adults with generalised myasthenia gravis (gMG), and is reportedly the first long-acting C5 complement inhibitor to demonstrate clinical improvement in patients with the disease.
What is gMG?
gMG is a rare and debilitating chronic, autoimmune and neuromuscular disease which leads to loss of muscle function and severe weakness in patients.
The disease operates by activating the complement cascade in the immune system in an uncontrollable manner, leading the body to attack healthy cells in the system.
An estimated 90,000 patients in the US currently suffer from the condition.
Ultomiris clinical trial
The FDA reportedly approved the treatment based on positive results from the pharmaceutical group’s CHAMPION-MG phase three trial, in which Ultomiris displayed superior results to the placebo in the company’s Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at week 26.
The MG-ADL is a patient-reported scale which assessed the ability of patients to perform daily activities during the trial.
The treatment worked as a long-lasting C5 complement inhibitor, which offered complete and sustained inhibition to the C5 protein in the terminal complement cascade.
Ultomiris is administered to patients intravenously once per eight weeks in adult patients, after a loading dose.
AstraZeneca commented that the most common adverse reactions in participants were upper respiratory tract infection and diarrhoea.
The results from the CHAMPION-MG trial were published in the NEJM Evidence journal and presented at the American Academy of Neurology Annual Meeting in April 2022.
The European Union and Japan are reportedly considering submissions by AstraZeneca for the treatment, along with a selection of other health authorities.
“gMG takes a physical and emotional toll on those living with the disease,” said Myasthenia Gravis Foundation of America CEO Samantha Masterson.
“We are grateful for continued innovation and research into new treatment and dosing options to meet the needs of more patients and reduce the treatment burden.”
“With the approval of Ultomiris, we’re excited that MG patients now have another option to consider as part of their personalised treatment strategies that may offer more convenience and improve muscle weakness.”
