A year after the FDA issued Celgene a refuse-to-file letter for its multiple sclerosis drug ozanimod, the biotech is filing for regulatory approval in the European Union, with plans to refile in the U.S. this month.
Celgene picked up ozanimod, an S1P receptor modulator, back in 2015, in its $7.2 billion acquisition of Receptos. It is seeking approval in the EU for relapsing-remitting multiple sclerosis (MS) and in the U.S. for relapsing forms of the disease.
Celgene had reckoned that a better safety profile than Novartis’ Gilenya—particularly with regard to "liver toxicities, QT prolongation and bradycardia”—boded well for the drug’s approval, but a substandard filing resulted in a refuse-to-file letter in February 2018.
The FDA sent a letter after determining that the nonclinical and clinical pharmacology information Celgene provided were not up to snuff. The setback added a year to ozanimod’s timeline, delaying the launch of a potential blockbuster with peak sales around $5 billion per year.
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At the time, management explained that the letter was unrelated to potential concerns about S1P receptor modulators and said it thought it could use existing activities to address some of the agency’s feedback. But the incident also raised questions about Celgene’s credibility.
“This just adds even more uncertainty and credibility questions, which is not helpful right now given Celgene is in a period where they need to instill confidence after the recent disappointments,” wrote Jefferies Analyst Michael Yee at the time.
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In June 2018, Nadim Ahmed, Celgene’s president of haematology and oncology, pinned the blame for the filing on Receptos. He acknowledged that such refusal letters are rare for companies like Celgene and framed the dip in standards as a lesson in the need to integrate acquisitions.
“[The FDA] kinda said ‘what happened guys, this isn’t what we usually expect from Celgene?’ And we had to say, you know, ‘mea culpa it’s on us’,” Ahmed said. “I think that 99% of folk at Celgene wouldn’t have submitted, but we had Receptos out on the West Coast and, for whatever reason, the decision was made to submit.”