Uplizna now approved in Canada to treat adults with NMOSD
Therapy indicated for patients positive for disease-driving AQP4 antibodies
Health Canada has approved Uplizna (inebilizumab-cdon) to treat adults in the country with neuromyelitis optica spectrum disorder (NMOSD) who test positive for disease-driving anti-aquaporin-4 (AQP4) antibodies.
The decision adds Canada to the list of nations where the medication is now available.
The intravenous or into-the-vein infusion, developed by Horizon Therapeutics, now part of Amgen, was first approved in the U.S. in 2020 for the same indication. It more recently was given the green light for use in Japan and Brazil, and across Europe.
“Today’s approval of Uplizna marks a significant milestone for adults living with NMOSD in Canada, bringing a new, targeted treatment option to those living with this devastating disease,” Matt McCarthy, Horizon’s general manager in Canada, said in a press release.
After start-up doses, Uplizna is given to patients every 6 months
NMOSD occurs when the body’s own antibodies attack primarily the optic (eye) nerve and spinal cord, causing inflammation. Each attack causes new damage that builds up over time, potentially leading to loss of vision and sensation, muscle weakness, and inability to control the bladder.
“Just a single NMOSD attack can have a life-altering impact, including pain, debilitation and irreversible vision loss,” McCarthy said.
“We are committed to bringing new medicines to people living with rare and challenging diseases around the world, and today’s announcement is an exciting milestone in that effort,” McCarthy added.
Due to its varied symptoms, which can mimic other diseases, NMOSD can be challenging to diagnose. As such, the autoimmune disorder may not be readily identified, leading to a delay in diagnosis and proper treatment.
“NMOSD has historically been misdiagnosed for multiple sclerosis (MS), which can delay proper treatment and worsen outcomes, especially if treated with MS drugs,” said Mark Freedman, MD, who directs the multiple sclerosis research unit at Ottawa Hospital, in Canada.
According to Freedman, who doubles as senior scientist at Ottawa Hospital Research Institute and a professor of neurology at the University of Ottawa, “we can now accurately diagnose NMOSD, which is distinctly different from MS and warrants specific treatment.”
Uplinza is an important new treatment option that provides physicians and patients living with NMOSD a therapy with proven efficacy, a favorable safety profile and a twice-yearly maintenance dosing schedule.
Antibodies, including those that zero in on the body’s own cells, are produced by a type of immune cells called B-cells. Uplizna attaches to B-cells and helps to destroy them. That’s expected to ease the symptoms of NMOSD and prevent new attacks from happening.
After an initial round of two start-up doses, given two weeks apart as an infusion directly into the bloodstream, treatment with Uplizna follows a maintenance dosing schedule. Single infusions are given every six months.
“Uplinza is an important new treatment option that provides physicians and patients living with NMOSD a therapy with proven efficacy, a favorable safety profile and a twice-yearly maintenance dosing schedule,” Freedman said.
Its approval was based on data from a pivotal Phase 2/3 clinical trial called N-MOmentum (NCT02200770), in which Uplizna outperformed a placebo at reducing the risk and severity of relapses of relapses, or flare-ups.
The N-MOmentum trial, involving 230 adults with NMOSD, showed that, among those with anti-AQP4 antibodies, 87.6% of patients on Uplizna remained relapse-free over six months after an infusion. That was compared with 56.6% of those given the placebo.
Sumaira Ahmed, founder and executive director of The Sumaira Foundation, a nonprofit organization, has lived with NMOSD for more than a decade.
“As a patient living with this rare disease for ten years, I know first-hand how important it is to have multiple treatment options available. As a patient advocacy leader, I have seen how they can transform the lives of fellow patients,” Ahmed said.
Ahmed welcomed Uplizna’s approval.
“We at The Sumaira Foundation are excited to see new, clinically proven therapies becoming available to NMOSD patients in Canada that give them additional options for treatment and management of this rare but serious condition,” Ahmed said.