DUBLIN– Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for UPLIZNA® (inebilizumab) as a monotherapy for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+). UPLIZNA has been granted orphan designation by the European Commission (EC). That designation is subject to additional review by the Committee for Orphan Medicinal Products.
UPLIZNA has not been approved for commercial use in the European Union (EU). Based on the CHMP recommendation, a decision by the EC, if granted, would authorize marketing approval in the EU. This centralized marketing authorization would be valid in all EU Member States, as well as in Iceland, Liechtenstein and Norway.
“Today’s positive CHMP opinion is a significant milestone in Horizon’s global growth and long-term commitment to address critical needs of those living with rare, autoimmune and severe inflammatory diseases,” said Tim Walbert, chairman, president and chief executive officer, Horizon. “In Europe, people living with NMOSD currently have very limited targeted treatment options and we look forward to advancing care for these patients who live daily with unpredictable attacks, pain and disability.”
UPLIZNA was approved by the FDA in June 2020 and by the Japanese Ministry of Health, Labour and Welfare in March 2021 as a targeted CD19 B-cell depleting antibody for adult patients with AQP4-IgG+ NMOSD, to reduce the risk of attacks. UPLIZNA is the only approved NMOSD therapy in the U.S. that has demonstrated a clinically relevant and durable effect on delaying worsening of disability, with a significant reduction in hospitalization. In Japan, Mitsubishi Tanabe Pharma Corporation has the rights for development and commercialization of UPLIZNA. Long-term UPLIZNA treatment has been shown to be well tolerated and provide a sustained reduction in NMOSD attack risk for four or more years.1
NMOSD is a rare, severe autoimmune disease where the body’s defense cells (B-cells) start to attack the optic nerve, spinal cord and brain stem.2,3 NMOSD is often misdiagnosed as multiple sclerosis (MS) and primarily damages the optic nerve(s) and spinal cord, causing permanent blindness, muscle weakness and paralysis.4 NMOSD is characterized by unpredictable attacks and severe disability that often occurs following the first attack, accumulating with each subsequent relapse.5 Preventing these attacks is the primary goal for disease management.6 UPLIZNA works by depleting B-cells in a targeted manner, and patients need only take it once every six months, via infusion, following an initial two treatments spaced two weeks apart.7
AQP4 is a membrane protein that is predominantly found in the central nervous system. It has a protective effect by regulating the water homeostasis, a self-regulating process, to maintain the stability of the physiological function in the body.8 A defining feature of NMOSD (seen in approximately 80% of patients) is the presence of serum autoantibodies which work against AQP4, leading to severe attacks in the central nervous system, resulting in substantial and often permanent disability.9
Globally, the prevalence of NMOSD is approximately 0.5–4/100,000 people.10, 11 Europe has an estimated disease population of at least 7,300 patients, 75-80% of which are AQP4-IgG+. Each year, approximately 370 new patients in Europe are diagnosed with NMOSD.12
“NMOSD is a very devastating and serious condition where symptoms become worse over time and are followed by periods of less severe symptoms that do not completely cease, so my patients never know what to expect,” said Professor Friedemann Paul, M.D., Charité-Universitätsmedizin Berlin, NeuroCure Clinical Research Center, NCRC Research Group, „Clinical Neuroimmunology“. “During a relapse, damage to the optic nerves and/or spinal cord can lead to accumulating disability, therefore, preventing attacks is critical to a good long-term outcome. There continues to be a high need for new treatments, specifically developed for NMOSD, that can help to ease symptoms and prevent future relapses. This is why the news of the positive CHMP recommendation for UPLIZNA brings such hope to the NMOSD patient community.”
The CHMP positive opinion was granted based on the data from the N-MOmentum clinical development program (NCT02200770), which found that UPLIZNA monotherapy reduced the risk of relapse by 77% compared to placebo in AQP4-IgG+NMOSD adult patients.7
