DUBLIN– Horizon Therapeutics plc (Nasdaq: HZNP) today announced the European Commission (EC) approval of UPLIZNA® (inebilizumab) as monotherapy for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+), following the positive opinion adopted by the Committee for Medicinal Products for Human Use of the European Medicines Agency on 11th November, 2021.
“NMOSD is a devastating disease with unpredictable attacks, cumulative and often irreversible damage and potential loss of vision and motor function, causing profound uncertainty for patients,” said Vikram Karnani, executive vice president and president, international, Horizon. “Today’s approval of UPLIZNA marks a significant milestone for patients in Europe and for Horizon, bringing a new, targeted treatment option to people living with NMOSD. As we continue our global expansion, we remain focused on bringing breakthrough medicines – and hope – to people living with challenging diseases. Across offices and manufacturing facilities on four continents, we deliver impact that goes beyond our medicines.”
People impacted by NMOSD live with unpredictable attacks; 90% will experience repeat attacks within five years of an initial attack.1 Even a single NMOSD attack can result in significant, irreversible damage, with each subsequent attack building upon the damage caused by the previous.2 This damage is caused when CD19+-expressing B-cell lymphocytes (plasmablasts and some plasma cells) secrete AQP4-IgG, triggering an escalating autoimmune reaction. Depletion of CD19+ B-cells has proven effective at halting inflammation, lesion formation and astrocyte loss. As a targeted CD19 B-cell depleter, UPLIZNA offers a unique mode of action developed specifically to induce broad, deep and durable B-cell depletion and prevent attacks. Maintaining B-cell depletion and preventing attacks are key to limiting cumulative disability within NMOSD.3,4,5
In the N-MOmentum pivotal clinical trial (2014-000253-36), the largest NMOSD trial to date, UPLIZNA demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial loading doses. Additionally, 89% of patients in the AQP4-IgG+ group remained relapse-free during the six-month period post-treatment and more than 83% of patients on treatment remained attack free for at least four years.3,4
“We have made great strides in understanding the pathogenesis of NMOSD and in identifying new, effective therapies for its treatment, such as UPLIZNA, which can be transformative for patients in Europe who live with the devastating effects of this disease,” said Professor Friedemann Paul, M.D., Charité-Universitätsmedizin Berlin, NeuroCure Clinical Research Center, NCRC Research Group, Clinical Neuroimmunology. “Just a single attack can cause life-altering impact, pain and debilitation, including potential vision loss. It is paramount we’re able to diagnose and treat this disease early to prevent cumulative damage and permanent disability for patients.”
Globally, the prevalence of NMOSD is approximately 0.5–4/100,000 people,6,7 and women are nine times more likely to be impacted than men.8 Consequences of this disease extend beyond the clinical impact, including physical, functional and psychological impact on patients’ quality of life.9,10 In Europe, it is estimated that at least 7,300 people live with NMOSD,11 approximately 80% of whom are AQP4-IgG+.12 Each year, approximately 370 new patients in Europe are diagnosed with the disease.11
The EC approval of UPLIZNA results in the granting of a centralised marketing authorisation, which is valid in all EU Member States, as well as in Iceland, Liechtenstein and Norway. Horizon will work with local health authorities to bring UPLIZNA to patients in a number of countries across Europe. Germany will be the first country where Horizon initiates commercialisation efforts.
UPLIZNA was approved by the U.S. Food and Drug Administration (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.
Mitsubishi Tanabe Pharma Corporation has the rights to develop and commercialise UPLIZNA in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, Philippines, Singapore and Taiwan. Hansoh Pharmaceutical Group Company Limited, another strategic partner to Horizon, has also recently received manufacturing and marketing approval from National Medical Products Administration of the People’s Republic of China for UPLIZNA.
