Ozanimod accepted for priority review by FDA for the treatment of ulcerative colitis
The priority review is based on results from a placebo-controlled Phase 3 trial evaluating ozanimod as a therapy for moderately to severely active ulcerative colitis.
February 03, 2021
LA JOLLA, CA—An immune-modulating therapy invented at Scripps Research has been accepted for filing with priority review by the U.S. Food and Drug Administration (FDA). The drug, ozanimod, which is being developed by Bristol Myers Squibb under the name Zeposia, gained FDA and European Commission approval last year for multiple sclerosis.
Bristol Myers Squibb this week announced the priority review, based on the results of True North, a pivotal, placebo-controlled Phase 3 trial evaluating ozanimod as a single daily dosed oral therapy for adults and children over the age of 12 years with moderately to severely active ulcerative colitis.
The priority review designation, as a result of the redemption of a Priority Review Voucher by Bristol Myers Squibb, is a significant additional investment in the development of Zeposia accelerating its potential launch for this next indication. The FDA will take action on an application within six months from initial submission, compared with the regular ten months under standard review. This means that ozanimod could be approved by FDA on or before 30 May 2021 and become the first-in-class sphingosine 1-phosphate receptor modulator for ulcerative colitis.
“Results like this are gratifying to see, both for the medical community and for patients around the world who are in need of better options to manage this unpredictable disease,” says Hugh Rosen, MD, PhD, who invented ozanimod along with fellow Scripps Research professor Edward Roberts, PhD, and their laboratory colleagues. “Based on the outcome of this pivotal trial and the ongoing review, I am confident this approach will make a real difference for patients with an oral agent of outstanding efficacy and safety.”
In the randomized and blinded clinical study, more than 600 patients taking ozanimod were compared with more than 300 patients on placebo. A far higher share of patients who took ozanimod for 10 weeks experienced remission, meaning that symptoms decreased to the point that they were mostly absent or gone. At week 52, patients on the drug also maintained their remission at a higher rate. In addition, the study met secondary endpoints for clinical response—noting a marked decrease in disease activity—and endoscopic improvement, which refers to healing of the mucosal tissue in the colon and rectum.
Ulcerative colitis, part of a group of conditions known as inflammatory bowel diseases, is the result of the immune system’s overactive response. It is a relapsing, chronic autoimmune disease of the large intestine, in which the lining of the colon becomes inflamed and develops tiny open sores, or ulcers. Ozanimod works by acting on certain types of immune cells called lymphocytes that are centrally involved in the autoimmune attack on the large intestine. It binds to receptors on the cells’ surface, diminishing the immune attack. If approved, ozanimod will be the first drug of its class for the treatment of ulcerative colitis.
Ozanimod is also in late-stage clinical trials for the treatment of Crohn’s disease, another type of inflammatory bowel disease. While ulcerative colitis affects the colon and rectum, Crohn’s disease may act on any part of the gastrointestinal tract and also affect the entire thickness of the bowel wall.
The fundamental 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 $7.2 billion. Celgene was acquired by Bristol Myers Squibb in 2019.
Additional molecules developed by Rosen and Roberts at Scripps Research are currently in phase 2 clinical trials for major depressive disease and anxiety, and phase 1 studies for treatment of autism.
For more information, contact press@scripps.edu