FDA Greenlight’s Viela Bio’s Uplizna for Rare Neuroinflammatory Autoimmune Disorder

FDA Greenlight’s Viela Bio’s Uplizna for Rare Neuroinflammatory Autoimmune Disorder

The U.S. Food and Drug Administration (FDA) approved Viela Bio’s Uplizna (inebilizumab-cdon) for adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-AQP4 antibody positive. About 80% of all NMOSD patients test positive for anti-AQP4 antibodies.

NMOSD has symptoms similar to multiple sclerosis (MS) and at one time was considered a variation of MS. But the disease typically affects mostly the eyes and the spinal cord, compared to MS, which also affects the brain. The risk of being disabled is also greater with NMOSD.

NMOSD is an autoimmune disorder, where the immune system attacks a protein called aquaporin 4, which is found on the surface of astrocytes in the brain, spinal cord, and optic nerves. This damage is believed to cause demyelination. In some patients with NMOSD, the immune system targets a different protein, myelin oligodendrocyte glycoprotein (MOG), which is found on the outer layer of myelin.

Symptoms include inflammation of the optic nerve, which can cause eye pain and dim, blurred, or loss of vision. Sometimes the limbs are affected, with individuals temporarily losing sensation, muscle spasms, weakness and paralysis. They may lose control of the bladder and bowel. Intractable hiccups and nausea and vomiting also may occur.

“NMOSD is an extremely challenging disease to treat,” said Bruce Cree, the lead investigator for the N-Momentum trial and professor of Clinical Neurology at the University of California San Francisco Weill Institute for Neurosciences. “Patients experience unpredictable attacks that can lead to permanent disability from blindness and paralysis. In addition, each subsequent attack may result in a cumulative worsening of disability. In the pivotal N-Momentum trial, Uplizna—a humanized CD19-directed monoclonal antibody—significantly reduced the risk of attacks and also reduced hospitalized when given as a monotherapy.”

The drug had received both Breakthrough Therapy and Orphan Drug designations from the FDA. In the N-Momentum trial, 213 anti-AQP4 antibody positive patients and 17 anti-AQP4 antibody negative patients met the primary endpoint of statistically significant reduction in risk of NMOSD attacks. In the trial, 89% of the anti-AQP4 antibody positive group were relapse-free during the six-month period after treatment, compared to 58% of the placebo group.

The drug also showed statistically significant benefits in key secondary endpoints, including decreases in NMOSD-related hospitalizations. The drug had a favorable safety and tolerability profile. The most common adverse events were urinary tract infection, nasopharyngitis (stuffy nose), infusion reaction, stiff joints and headache.

The study was concluded early after an independent Data Monitoring Committee recommended it based on evidence of efficacy.

“Until recently, patients with NMOSD had no FDA-approved treatment options,” said Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “Uplizna now represents the second approved therapy for these patients within the past year. We continue to remain highly committed to the development of additional safe and effective drugs for this rare and devastating disease.”

The other approved treatment is Alexion Pharmaceuticals’ Soliris (eculizumab). Although Viela Bio has not publicly announced prizing for Uplizna, Alexion’s Soliris runs about $500,000 per year.

Uplizna is also in a Phase II trial in kidney transplant desensitization and another Phase III trial is planned for myasthenia gravis and a Phase IIb trial planned in IgG4-related disease.

“We are proud that Viela Bio’s first approved medicine has the potential to help thousands of patients with NMOSD, a progressive and debilitating neuroinflammatory disease,” said Bing Yao, Biela Bio’s chief executive officer. “We are incredibly grateful to the patients, families and care partners who participated in and supported our research.”

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