A stamp of approval for ozanimod

LA JOLLA, Calif.—The U.S. Food and Drug Administration (FDA) has announced its approval of ozanimod, an immune-modulating therapy invented at Scripps Research, for the treatment of adults with relapsing forms of multiple sclerosis.

In patient studies supporting ozanimod’s New Drug Application, those who took the once-daily oral medicine experienced significantly less disease progression — including fewer relapses, and preservation of the brain from atrophy — than those who received standard care, with very few side effects. The drug, which has been licensed to Bristol Myers Squibb, will be sold under the trade name Zeposia.

“Today’s FDA approval of ozanimod is a celebratory milestone for the multiple sclerosis community, which is in need of new, intelligent drug interventions to help patients control the progression of their disease,” said Hugh Rosen, M.D., Ph.D., who invented ozanimod along with fellow Scripps Research professor Edward Roberts, Ph.D., and their laboratory colleagues.

The discoveries that led to ozanimod were reported by Rosen, Roberts and their Scripps Research colleagues in a series of papers from 2002 to 2008. In 2009, Scripps Research licensed ozanimod to biotechnology startup Receptos, which Celgene purchased in 2015 for $7.3 billion. Celgene was acquired by Bristol Myers Squibb in 2019.

Ozanimod works by acting on certain types of immune cells called lymphocytes that are centrally involved in the autoimmune attack on myelin sheath. It binds to receptors on the cells’ surface, keeping them from reaching the brain. As a result, the number of activated lymphocytes is decreased, diminishing the immune attack. Nearly a million people are living with multiple sclerosis in the U.S., according to the National Multiple Sclerosis Society, and approximately 85 percent of those patients are diagnosed with the relapsing forms of the disease that ozanimod is designed to treat.

Ozanimod is also being studied as a treatment for forms of inflammatory bowel disease. The drug is in advanced clinical development for adults and children with moderate-to-severe ulcerative colitis and Crohn’s disease, for which it is a first-in-class treatment.

The ozanimod approval for multiple sclerosis is the latest in a string of FDA-approved drugs to originate from Scripps Research’s laboratories, following on the most recent approval of tafamidis for the rare but often fatal heart disease known as transthyretin amyloid cardiomyopathy (ATTR-CM).