Summary: Ozanimod, an immune-modulating medication developed at Scripps Research, has received FDA approval for the treatment of adults with relapsing forms of multiple sclerosis. The drug prevents certain lymphocytes from entering the brain by binding to receptors on their surface, reducing the number of activated immune cells that drive attacks on the nervous system.
Source: Scripps Research Institute
The U.S. Food and Drug Administration has approved ozanimod, an immune-modulating therapy invented at Scripps Research, for the treatment of adults with relapsing forms of multiple sclerosis. The drug is also in advanced clinical development for adults and children with moderate-to-severe ulcerative colitis and Crohn’s disease.
Clinical trials submitted to the FDA showed that patients taking once-daily oral ozanimod experienced significantly less disease activity than those receiving standard care. Trial participants had fewer relapses and showed better preservation of brain volume, with a favorable tolerability profile and relatively few adverse effects. The therapy, licensed to Bristol Myers Squibb, will be marketed under the brand 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,” says Hugh Rosen, MD, PhD, who invented ozanimod along with fellow Scripps Research professor Edward Roberts, PhD, and their laboratory colleagues.
Nearly 1 million people in the United States live with multiple sclerosis, and roughly 85 percent of those diagnosed have relapsing forms of the disease that ozanimod targets. Relapsing MS is characterized by episodes of new or worsening neurological symptoms followed by periods of partial or full recovery. These flare-ups are driven by immune cells mistakenly attacking the protective myelin sheath that surrounds nerve fibers in the brain and spinal cord.
Damage to myelin interrupts the efficient transmission of electrical signals between the brain and the rest of the body, producing symptoms that range from numbness, tingling, and bladder dysfunction to visual disturbances and muscle weakness or paralysis. Therapies that reduce the frequency and severity of relapses can help preserve neurological function and slow disability progression.
Ozanimod reduces immune-driven damage by acting on a subset of immune cells called lymphocytes, which play a central role in the autoimmune attack on myelin. By binding to receptors on the surface of these cells, ozanimod prevents them from migrating into the central nervous system. This lowers the number of circulating activated lymphocytes available to initiate or sustain inflammatory attacks on nerve tissue, thereby reducing disease activity.
The foundational discoveries that led to ozanimod’s development were published by Rosen, Roberts and colleagues from 2002 to 2008. In 2009, Scripps Research licensed ozanimod to the biotechnology company Receptos. Receptos was later acquired by Celgene in 2015, and Celgene subsequently became part of Bristol Myers Squibb in 2019. Through these partnerships, ozanimod advanced through clinical development and regulatory review to reach patients.
Beyond multiple sclerosis, ozanimod is being evaluated as a treatment for inflammatory bowel diseases. Late-stage clinical trials are underway for both ulcerative colitis and Crohn’s disease, conditions in which immune-driven inflammation damages the gastrointestinal tract. If approved for these indications, ozanimod would represent an additional treatment option for patients with moderate-to-severe disease.
The FDA approval of ozanimod adds to a growing list of therapies that originated in Scripps Research laboratories and later reached the clinic. Most recently, the institution celebrated approval of tafamidis for ATTR-CM, a rare but serious heart condition. Scripps Research has played a role in discovering or developing medications for a wide range of diseases, addressing unmet medical needs across areas such as autoimmune disorders, oncology, hematology, and infectious disease.
Researchers at Scripps continue to advance additional drug candidates discovered in their laboratories. Other molecules developed by the teams led by Rosen and Roberts are currently in clinical testing for neuropsychiatric and developmental conditions: some candidates are in phase 2 trials for major depressive disorder and anxiety, while others are in early phase 1 studies exploring potential treatments for autism.
“There are hardly any other academic institutions in the world that have the multidisciplinary expertise to discover a new disease‑modifying compound and generate clinical data in support of its development,” Rosen notes, emphasizing the unique translational capabilities that helped move ozanimod from laboratory discovery to an approved therapy.
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Scripps Research Institute
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