WASHINGTON -- Intravenous inebilizumab (Uplizna) won (NMOSD), the FDA announced late Thursday.
The anti-CD19, B-cell depleting monoclonal antibody was approved to treat adult NMOSD patients positive for anti-aquaporin-4 (AQP4) antibodies. NMOSD is a rare, severe, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, and brain stem.
Inebilizumab is the second treatment approved for NMOSD. The first was eculizumab (Soliris), approved in 2019.
"Until recently, patients with NMOSD had no FDA-approved treatment options," said Billy Dunn, MD, director of the Office of Neuroscience in the agency's Center for Drug Evaluation and Research. "We continue to remain highly committed to the development of additional safe and effective drugs for this rare and devastating disease."
Most NMOSD patients produce antibodies that target the water-channel protein AQP4. Binding of anti-AQP4 antibodies to central nervous system cells appears to trigger NMOSD attacks.
Disability in NMOSD is due to attacks, with each attack leading to more damage. Potentially irreversible blindness and paralysis may occur, and patients may experience loss of sensation, bladder and bowel dysfunction, nerve pain, and respiratory failure.
The FDA based its decision on the pivotal clinical trial. "Multiple lines of evidence suggest that NMOSD is a B-cell mediated disorder," said Bruce Cree, MD, PhD, of the University of San Francisco Medical Center, who presented trial results at the 2019 American Academy of Neurology annual meeting. CD19 is expressed widely throughout B-cell development in NMOSD, he noted.
In N-MOmentum, 213 of 230 patients had antibodies against AQP4. Inebilizumab by 77% in patients positive for anti-AQP4 antibodies, relative to placebo, but showed no evidence of benefit in patients negative for anti-AQP4 antibodies. The most common adverse reactions in the trial were urinary tract infection, headache, joint pain, nausea, and back pain.
Prescribing information includes warnings about infusion reactions, hypogammaglobulinemia, potential increased risks of infection and progressive multifocal leukoencephalopathy (an inflammatory brain condition caused by reactivation of latent JC virus infection), and potential reactivation of hepatitis B and tuberculosis, the FDA said.
Pregnant women should not take inebilizumab because it may cause harm to a developing fetus or newborn baby. Clinicians should inform reproductive-age females about using effective contraception during inebilizumab treatment and for 6 months after the last dose. Vaccination with live-attenuated or live vaccines is not recommended during treatment and should be administered at least 4 weeks before starting inebilizumab, the agency added.
The drug is administered as a twice-a-year maintenance regimen following initial doses. Drug maker Viela Bio expects the this month. A phase II trial with inebilizumab in kidney transplant desensitization is ongoing, and a phase II trial in myasthenia gravis and phase IIb trial in IgG4-related disease are planned, the company said.