FDA News Release
FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord
Second FDA Approved Therapy for Neuromyelitis Optica Spectrum Disorder Offers Patients Additional Treatment Option
- For Immediate Release:
The U.S. Food and Drug Administration today approved Uplizna (inebilizumab-cdon) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients with a particular antibody (patients who are anti-aquaporin-4 or AQP4 antibody positive). NMOSD is a rare autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. Uplizna is only the second approved treatment for the disorder.
“Until recently, patients with NMOSD had no FDA-approved treatment options,” said Billy Dunn, M.D., 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.”
In patients with NMOSD, the body’s immune system mistakenly attacks healthy cells and proteins in the body, most often those in the optic nerves and spinal cord. Individuals with NMOSD typically have attacks of optic neuritis, which causes eye pain and vision loss. Individuals also can have attacks resulting in transverse myelitis, which often causes numbness, weakness, or paralysis of the arms and legs, along with loss of bladder and bowel control. Most attacks occur in clusters, days to months to years apart, followed by partial recovery during periods of remission. Approximately 50% of patients with NMOSD have permanent visual impairment and paralysis caused by NMOSD attacks. According to the National Institutes of Health, women are more often affected by NMOSD than men and African Americans are at greater risk of the disease than are Caucasians. Estimates vary, but NMOSD is thought to impact approximately 4,000 to 8,000 patients in the United States.
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.
The effectiveness of Uplizna for the treatment of NMOSD was demonstrated in 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% 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 (arthralgia), nausea and back pain. Women who are pregnant should not take Uplizna because it may cause harm to a developing fetus or newborn baby. The FDA advises health care professionals to inform females of reproductive age to use effective contraception during treatment with Uplizna and for six months after the last dose. Vaccination with live-attenuated or live vaccines is not recommended during treatment and should be administered at least four weeks prior to initiation of Uplizna.
Uplinza received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted approval of Uplizna to Viela Bio.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
- Kristin Jarrell