Ultomiris (ravulizumab) approved for the treatment of adults with neuromyelitis optica spectrum disorders (NMOSD) in the EU
17.05.2023 12:42
Ultomiris has the potential to prevent relapse events and improve treatment outcomes, changing the therapeutic landscape for patients with AQP4 antibody-positive NMOSD
AstraZeneca, together with Alexion, AstraZeneca Rare Disease, announced that Ultomiris (ravulizumab) is the first and only long-acting C5 complement inhibitor for the treatment of adult patients with aquaporin 4 (AQP4) antibody-positive neuromyelitis optica spectrum disorders (NMOSD) approved in the European Union (EU).
became. [1]
The European Commission's approval follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is based on the results of the phase III CHAMPION-NMOSD study, which was published online in Annals of Neurology and as an abstract at the annual meeting 2023 awards from the American Academy of Neurology. [2] Ultomiris was compared with the external placebo arm of the Soliris (eculizumab) pivotal PREVENT clinical trial.
An independent panel confirmed that Ultomiris met the primary endpoint, defined as time to first attack. With Ultomiris, no relapses occurred during a median treatment period of 73 weeks (reduction in relapse risk: 98.6 %, hazard ratio (95 % CI): 0.014 (0.000; 0.103), p < 0.0001), with freedom from relapses in the ongoing follow-up lasted a median of 90 weeks. [1]
NMOSD are rare and debilitating autoimmune diseases that affect the central nervous system (CNS), including the spinal cord and optic nerves. [3-5] Most NMOSD patients suffer from unpredictable relapses that are accompanied by new symptoms or a worsening of existing neurological symptoms. The attacks are usually severe and recurring and can lead to permanent impairment. [6-8] The diagnosis prevalence of adults with NMOSD in the EU is estimated to be approximately 5,000. [9]
Prof. Dr. med. Orhan Aktas, Professor at the Department of Neurology at the University Hospital Düsseldorf, emphasized: “Effective treatment of NMOSD depends on reducing relapse events, because even a single relapse can lead to irreversible impairments, so that those affected can no longer move, speak or can take care of yourself. The approval of Ultomiris in Europe offers patients and physicians a new and long-acting therapy with an unprecedented reduction in relapse risk. In the approval study, there were no relapses with Ultomiris at a dosage interval of every eight weeks.”
“Just four years after the first approval of a treatment for NMOSD in the EU, we are very pleased to once again advance the treatment of this rare neurological disease with Ultomiris,” said Graham Skarnvad, General Manager, Alexion Pharma Germany. “The approval is the result of intensive work and collaboration with the NMOSD community, including patients, caregivers and healthcare professionals who participated in the CHAMPION-NMOSD study. With the approval, we see ourselves strengthened in our efforts to sustainably change the lives of those affected by NMOSD and give them a better quality of life.”
Overall, the safety and tolerability of Ultomiris in the CHAMPION NMOSD study were consistent with previous clinical trial and real-world data. No new safety alerts were observed. The most common adverse events (AEs) were COVID-19, headache and back pain, arthralgia and urinary tract infections. All COVID-19 cases were not serious and were classified as not healthcare-related. [2]
Marketing authorization applications for Ultomiris for the treatment of NMOSD are also currently under review by regulatory authorities in several countries including the United States (US) and Japan.
credentials
1. Ultomiris, technical information, May 2023.
2. Pittock SJ, Barnett M et al. Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial. Presentation at the American Academy of Neurology Annual Meeting, April 23, 2023; Presentation S5.002.
3. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805-815. 4. Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologist. 2007;13(1):2-11.
5. Hamid SHM, Whittam D, Mutch K et al. What proportion of AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG positive? A cross-sectional study of 132 patients. J Neurol. 2017;264(10):2088-2094.
6. Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options Neurol. 2008;10(1):55-66.
7. Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan. Brain. 2012;135(6):1834-1849.