The U.S. Food and Drug Administration stunned the company with a Refusal to File letter regarding its New Drug Application (NDA) for multiple sclerosis treatment ozanimod.
Shares of Celgene fell more than 7 percent in late Tuesday trading after the U.S. Food and Drug Administration stunned the company with a Refusal to File letter regarding its New Drug Application (NDA) for multiple sclerosis treatment ozanimod.
Celgene’s announcement was short on details. In its late-afternoon statement, Celgene said the FDA the company that the “nonclinical and clinical pharmacology sections” in the NDA were insufficient for the regulatory agency to move ahead in the approval process. Celgene said it will seek immediate guidance from the FDA to determine what kind of information it needs to move ahead.
Chief Medical Officer Jay Backstrom said the company remains confident in ozanimod’s clinical profile that was demonstrated in the “pivotal program” in relapsing forms of multiple sclerosis.
“We will work with the FDA to expeditiously address all outstanding items and bring this important medicine to patients,” Backstrom said in a statement.
The delay is certainly a blow to Celgene, which has been looking at ozanimod as a potential blockbuster drug that could become a key revenue driver for the company alongside multiple myeloma drug Revlimid. The company had pegged potential ozanimod revenue at more than $$ billion, particularly if the NDA for relapsing MS had been accepted.
Celgene gained ozanimod in the 2015 $7.2 billion acquisition of Receptos Inc. Ozanimod became a key focus for Celgene’s R&D in inflammatory bowel disease research, after another acquired drug, GED-0301, failed in a late-stage Crohn’s disease trial. The company discontinued the GED-0301 program for Crohn’s disease in October, a move that cost the company about $411 million. In addition to IBD ozanimod, a selective, sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator, showed promise with multiple sclerosis patients. In October Celgene shared positive Phase III results as a treatment for relapsing multiple sclerosis. Data showed ozanimod demonstrated superiority versus Biogen’s Avonex (interferon beta-1a) in reducing annualized relapse rates and MRI brain lesions. The SUNBEAM trial evaluated two doses of ozanimod (1 mg and 0.5 mg) in 1,346 patients with relapsing multiple sclerosis for at least one year. Trial data showed patients demonstrated a significant reduction in new or enlarging T2 lesions of 48 percent and 25 percent, respectively, over one year. Additionally, data showed a significant reduction in gadolinium-enhanced MRI lesions at one year. Data showed those reductions were 63 percent at 1 mg and 34 percent at .5 mg when compared to Avonex.
Celgene had spooked investors in October of last year after the company slashed its 2020 guidance but later won them back over after two acquisitions this year, including a $9 billion deal for Juno Therapeutics. Following news about the FDA’s letter investors once again got spooked and drove share prices down to $88, its lowest price since 2014.
