Lesson from FDA's panel meeting on Xarelto: Beware of "missingness." After a spirited debate over some missing study data, the advisory committee refused to back a big new use for the blood thinner. Between absent data on about 1,000 patients who withdrew from the trial and worries about its bleeding risks, Xarelto's bid for an expanded market went down.
The 6-4 vote doesn't doom Johnson & Johnson's ($JNJ) quest to market Xarelto for patients with acute coronary syndrome. The agency could still approve the new use, though it does tend to follow committees' advice. Further clouding the picture, however, is the fact that internal FDA assessments mirrored the panel's split vote. One reviewer recommended the new indication, while another questioned that pesky missing data.
"Were there not questions about loss to follow-up and missing data, it would have been a yes," pharmacist Alan Coukell, who voted no, told Reuters. "I want to see better evidence that this strategy of adding [Xarelto] ... is robustly better for the patient," said Steven Nissen of the Cleveland Clinic, who also voted against the new use.
"We appreciate the thoroughness of the committee's review, and will ensure the questions raised today are addressed with the FDA," said Paul Burton, a VP at J&J's Janssen unit. Leerink Swann analyst Seamus Fernandez predicted that the panel vote would delay the new use by as much as a year, Bloomberg reported.
Getting an ACS indication would help fuel Xarelto in its race against two other warfarin-alternative drugs: Pradaxa, from Boehringer Ingelheim, and Eliquis, from Bristol-Myers Squibb ($BMY) and Pfizer ($PFE). Both Xarelto and Pradaxa are approved to prevent stroke in patients with atrial fibrillation, while Eliquis awaits the FDA nod for that use. Xarelto is the only one of the three up for ACS approval. In ACS, it would compete with AstraZeneca's ($AZN) Brilinta and Eli Lilly's ($LLY) Effient.