The U.S. Food and Drug Administration (FDA) has approved Uplizna injection for intravenous use for the therapy of neuromyelitis optica spectrum disorder (NMOSD) that affects the spinal cord in the adult sufferers with an individual antibody (patients who are anti-aquaporin-4 or AQP4 antibody positive).
NMOSD is a rare autoimmune illness of the central nervous system that largely influences the optic nerves and spinal cord. Uplinza is just the second recommended therapy for the disease.
“Until lately, sufferers with NMOSD had no FDA-approved therapy options,” stated Billy Dunn, M.D., Director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research.
“Uplizna now serves the other recommended therapy for these sufferers within the past year. We remain highly engaged in the growth of additional safe and effective medications for this rare and destructive illness.”
In sufferers with NMOSD, the body’s immune method wrongly attacks healthy cells and proteins in the body, most often those in the optic nerves and spinal cord.
The sufferers with NMOSD typically have outbreaks of optic neuritis, which makes eye pain and vision loss. The sufferers can also have attacks resulting in transverse myelitis, which often causes numbness, dizziness, disease of the arms and legs, and loss of bladder and bowel control.
Most outbreaks occur in clusters, days to months to years apart, accompanied by partial recovery during periods of relief. Approximately 50% of sufferers with NMOSD have a persistent visual impairment, and disease NMOSD attacks produce them.
Placebo Therapy Society
According to the National Institutes of Health, women are more often influenced by NMOSD than men, and African Americans are at higher risk of the illness than are Caucasians. Estimates vary, but NMOSD is thought to affect roughly 4,000 to 8,000 patients in the United States.
NMOSD can be compared with immunoglobulins that bind to a protein named aquaporin-4 (AQP4). Binding of the anti-AQP4 antibody seems to activate other elements of the immune system, causing swelling and damage to the central nervous system.
The effectiveness of Uplizna for the therapy of NMOSD was confirmed in a clinical investigation of 230 adult cases that assessed the efficacy and safety of intravenous Uplizna.
In the trial, 213 of the 230 sufferers had antibodies against AQP4 (anti-AQP4 antibody positive). During the 197-day investigation, the risk of an NMOSD relapse in the 161 anti-AQP4 antibody-positive sufferers who were treated with Uplizna was decreased by 77% when connected to the placebo therapy society. There was no sign of a benefit in sufferers who were anti-AQP4 antibody negative.
Frequent Adverse Effects
The prescribing data for Uplizna includes a warning for infusion reactions, potential depletion of specific proteins (hypogammaglobulinemia), and potential heightened risk of infection – including Progressive Multifocal Leukoencephalopathy, and latent reactivation of hepatitis B and tuberculosis.
The most frequent adverse effects 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 child.
The FDA encourages health care experts to notify females of reproductive age to use effective contraception during therapy with Uplizna and for six months after the last dose.
The FDA Facts
Vaccination with live-attenuated or live vaccines is not supported during therapy and should be administered at least four weeks before the beginning of Uplizna. It sustained Orphan Drug designation, which gives incentives to assist and encourage the advancement of rare illnesses. The FDA, an agency within the U.S. Department of Health and Human Services, preserves the public health by ensuring the safety, effectiveness, and security of human and veterinary medicines, vaccines and other biological outcomes for human use, and medical devices.