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FDA Refuses to Review Celgene’s MS Drug, Says File “Insufficient”

Xconomy New York — 

Celgene’s bid to bring a potential blockbuster multiple sclerosis drug to the market was dealt a setback Tuesday after the FDA concluded the company’s application does not have enough information.

Summit, NJ-based Celgene (NASDAQ: CELG) said that it has received a “refusal to file” letter from the regulator regarding its submission for ozanimod. The company has been testing the pill as a treatment for patients who have a form of MS characterized by periodic relapses—flare-ups of neurological attacks. Refuse-to-file communications let a drug company know that the FDA has found deficiencies in a drug application. The notification gives a company the chance to address the deficiencies before a more serious “complete response letter,” which amounts to an FDA rejection.

Celgene said that the FDA found that the non-clinical and clinical pharmacology sections of its application were “insufficient to permit a complete review.” The company gave no other details in its announcement but added that it would ask the regulator for a meeting to find out what additional information is needed to refile. On a conference call Tuesday afternoon, Celgene executives indicated the company won’t need to run more clinical trials to appease the FDA. Management wouldn’t specify how long it might take to file a new approval application, however, according to a note from Mizuho Securities analyst Salim Syed.

Shares of Celgene dipped more than 6 percent to $89.28 per share in after-hours trading.

Ozanimod joined Celgene’s drug pipeline in 2015. It was part of Celgene’s $7.2 billion acquisition of San Diego-based Receptos. The drug was developed to treat immune-inflammatory diseases. Besides MS, the drug is in testing as a potential treatment for ulcerative colitis and Crohn’s disease. The drug is meant to have an anti-inflammatory effect and also activate cells in the central nervous system, which the company says could prevent neurological damage.

Celgene expects data from a Phase 3 trial in ulcerative colitis in late 2019. A Phase 3 study in ulcerative colitis should start this year. On the conference call, Celgene executives said neither of those studies are affected by the RTF letter, according to Syed.

In a research note, RBC Capital Markets analyst Brian Abrahams characterized ozanimod as one of the most important drugs in Celgene’s pipeline. The firm had projected the drug could reach $5 billion in peak sales as a treatment for MS and intestinal disorders. Abrahams called the setback “surprising,” and he added that it could worsen concerns about Celgene’s ability to continue to diversify its product line beyond core blood cancer drug lenalidomide (Revlimid).

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