Uplizna Approved in Europe for NMOSD Adults With Certain Antibodies
The European Commission has approved Uplizna (inebilizumab-cdon) to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who test positive for antibodies against aquaporin-4 (AQP4-IgG).
The approval covers all member states in the European Union, as well as Iceland, Liechtenstein, and Norway. Horizon Therapeutics, the company marketing Uplizna, will now be working with local governments to make the therapy commercially available, starting with Germany.
According to Horizon, at least 7,300 people live with NMOSD in Europe, of whom approximately 80% are AQP4-IgG-positive.
“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,” Vikram Karnani, executive vice president and international president of Horizon, said in a press release.
In NMOSD, antibodies made by the immune system drive an inflammatory attack that causes damage in the nervous system. Uplizna is designed to kill B-cells, the immune cells chiefly responsible for making antibodies, by targeting a protein on these cells called CD-19. The medication is administered twice a year via infusions into the bloodstream.
“We have made great strides in understanding the pathogenesis [disease development] 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 Friedemann Paul, MD, a researcher at Charité-Universitätsmedizin Berlin.
The therapy’s approval was supported by data from the clinical trial N-MOmentum (NCT02200770), in which 230 people with NMOSD were randomized to receive Uplizna or a placebo. Results showed that nearly nine in 10 participants given Uplizna were attack-free after 28 weeks, and the vast majority remained attack-free for up to four years.
Uplizna also was shown to ease pain, and in those participants who experienced attacks, Uplizna eased attack severity. A recent analysis indicated that it was similarly beneficial regardless of how many attacks participants had experienced prior to starting treatment.
“Just a single attack can cause life-altering impact, pain and debilitation, including potential vision loss,” Paul said. “It is paramount we’re able to diagnose and treat this disease early to prevent cumulative damage and permanent disability for patients.”
A committee of the European Medicines Agency recommended that Uplizna be approved in the EU last November. The therapy was approved in the U.S. in 2020, and in Japan in 2021. In Japan and several other regions in Asia, Uplizna is being commercialized in collaboration with Mitsubishi Tanabe Pharma.
“As we continue our global expansion,” Karnani said, “we remain focused on bringing breakthrough medicines — and hope — to people living with challenging diseases.”
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