One year after the FDA approved the first-ever treatment for a rare autoimmune condition that primarily affects the optic nerves and spinal cord, the agency has approved the second.
That second regulatory nod goes to Viela Bio (NASDAQ: VIE) drug inebilizumab (Uplizna) for adults with neuromyelitis optica spectrum disorder (NMOSD) who also have a particular antibody. The neuroinflammatory disease, which Viehla estimates affects about 10,000 people in the US, can cause eye pain, vision loss, and paralysis, as well as bladder and bowel dysfunction, nerve pain and respiratory failure.
Viela’s antibody drug is designed to target CD19, a protein broadly expressed on B cells; the company says research indicates the rare condition is likely B-cell mediated. The drug is delivered via infusion twice yearly after initial doses. It is intended for the roughly 80 percent of people with NMOSD who have antibodies that bind to a protein called anti-aquaporin-4 (AQP4), which is believed to prompt the immune system to attack patients’ central nervous system.
The FDA’s approval was based on data from a study that enrolled 230 patients, including 17 that tested negative for the antibody. Viehla last year reported the trial met its main goal of demonstrating a statistically significant reduction in risk of NMOSD attacks compared to patients who received a placebo. Eighty-nine percent of patients who tested positive for the antibody and received the drug had no relapses in the six months following treatment compared to 58 percent of the placebo group. There was no evidence of a benefit in patients who tested negative for the antibody.
Last June the FDA approved the first-ever NMOSD treatment, the Alexion Pharmaceuticals (NASDAQ: ALXN) drug eculizumab (Soliris). Eculizumab, which was previously approved in three other indications, comes with a black box warning on the drug’s label that cites the risk of life-threatening and fatal meningococcal infections.
Prescribing information for Viela’s inebilizumab warns health care professionals of the possibility of infusion reactions, depletion of proteins called gamma globulins, and risk of infection. Common side effects during the clinical trial included urinary tract infection, headache, joint pain, nausea, and back pain. The FDA says women who are pregnant should not take the drug, and that females of reproductive age should use contraception during treatment and for six months afterward. The label also warns against patients receiving live-attenuated or live vaccines during treatment.
The Viela drug is the first developed by the Gaithersburg, MD-based biotech, which in 2018 spun out of MedImmune, the AstraZeneca (NYSE: AZN) biologics R&D arm. AstraZeneca, which last year retired the MedImmune name, launched Viehla with its own financial backing plus more from a group of investors led by Boyu Capital, 6 Dimensions Capital, and Hillhouse Capital. At the time, inebilizumab, one of the six molecules from its early-stage inflammation and autoimmunity programs with which Viela debuted, was in Phase 2 testing.
The company plans to launch the drug this month.