FDA Approves Viela Bio’s Therapy for NMOSD
June 12, 2020
Rare Daily Staff
The U.S. Food and Drug Administration has approved Viela Bio’s Uplizna for the treatment of neuromyelitis optica spectrum disorder in adult patients with a particular antibody.
Neuromyelitis optica specrum disorder (NMOSD) is a rare, severe, neuro-inflammatory autoimmune disease that attacks the optic nerve, spinal cord and brain stem. In addition to potentially irreversible blindness and paralysis, patients may also experience loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure. It is estimated that there are approximately 10,000 people in the United States suffering from NMOSD.
NMOSD can be associated with antibodies that bind to a protein called aquaporin-4 (AQP4). Binding of the anti-AQP4 antibody appears to activate other components of the immune system, causing inflammation and damage to the central nervous system.
Bing Yao, CEO of Viela Bio, said the therapy has the potential to help thousands of patients with NMOSD.
Uplizna (inebilizumab-cdon) injection is approved for intravenous use in NMOSD patients who are AQP4 antibody positive, and is only the second approved treatment for the disorder, besides Alexion’s Soliris, which was approved last year.
The approval is based on a clinical study of 230 adult patients that evaluated the efficacy and safety of intravenous Uplizna. In the trial, 213 of the 230 patients had antibodies against AQP4 (anti-AQP4 antibody positive). During the 197-day study, the risk of an NMOSD relapse in the 161 anti-AQP4 antibody positive patients who were treated with Uplizna was reduced by 77 percent when compared to the placebo treatment group. There was no evidence of a benefit in patients who were anti-AQP4 antibody negative.
The prescribing information for Uplizna includes a warning for infusion reactions, potential depletion of certain proteins (hypogammaglobulinemia), and potential increased risk of infection – including Progressive Multifocal Leukoencephalopathy, and potential reactivation of hepatitis B and tuberculosis. The most common adverse reactions in the NMOSD clinical trial were urinary tract infection, headache, joint pain, nausea, and back pain.
Uplinza received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
Photo: Bing Yao, CEO at Viela Bio
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