New Treatment Approved by FDA for Rare Spinal Cord Disease

The FDA has approved inebilizumab-cdon (Uplizna, Viela Bio) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 or AQP4 antibody positive.

Inebilizumab-cdon is only the second approved treatment for the disorder, according to the FDA.

NMOSD is a rare autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. The body’s immune system mistakenly attacks healthy cells and proteins in the body, most often in the optic nerves and spinal cord, and can give patients attacks of optic neuritis.

Patients with NMOSD can also experience attacks resulting in transverse myelitis, which can cause numbness, weakness, and paralysis of the arms and legs, along with the 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.

The effectiveness of inebilizumab-cdon for the treatment of NMOSD was demonstrated in a clinical study of 230 adult patients that evaluated the efficacy and safety of intravenous inebilizumab-cdon. In the trial, 213 of the 230 patients had antibodies against AQP4, or were anti-AQP4 antibody positive.

The risk of an NMOSD relapse in the 161 anti-AQP4 antibody positive patients who were treated with inebilizumab-cdon was reduced by 77% when compared to the placebo treatment group in the 197-day study. There was no evidence of a benefit in patients who were anti-AQP4 antibody negative.

The most adverse reactions found in the NMOSD clinical trial were urinary tract infection, headache, joint pain, nausea, and back pain.

REFERENCE

FDA approves new therapy for rare disease affecting optic nerve, spinal cord. FDA. https://www.fda.gov/news-events/press-announcements/fda-approves-new-therapy-rare-disease-affecting-optic-nerve-spinal-cord?utm_campaign=061120_PR_FDA%20Approves%20Therapy%20for%20Rare%20Disease%20of%20Optic%20Nerve%2C%20Spinal%20Cord&utm_medium=email&utm_source=Eloqua. Published June 11, 2020. Accessed June 12, 2020.