It looks likely that Clovis Oncology’s non-small cell lung cancer drug rociletinib will hit a major setback in its quest for approval after failing to win the support of US Food and Drug Administration advisors.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 12-1 against approving the drug for patients with mutant epidermal growth factor receptor (EGFR) NSCLC who have been previously treated with an EGFR-targeted therapy and have the T790M mutation.

The Committee recommended that the regulator wait for results of TIGER-3, a randomised, controlled Phase III trial of rociletinib, before making a final decision on its approval. Patient enrollment is expected to complete in late 2018.

A key issue seems to be the drug’s safety profile. Even before the ODAC meeting, FDA staff reviewers were calling for “the inclusion of a Boxed Warning for the risk of QTc prolongation leading to Torsades de pointes”, as well as that labelling “describe ECG monitoring of QTc interval at baseline and periodically while receiving treatment with rociletinib”. 

There should also be information under the Warning and Precautions subsections for QTc prolongation, hyperglycaemia, interstitial ling disease/pneumonitis, pancreatitis, and cataracts, noted documents released ahead of the meeting.

The final word on drug still lies with the FDA, which doesn’t always follow the advice of its advisory committees. However, given the regulator’s concerns over the drug’s safety and efficacy, a rejection does currently seem likely before the target action date of June 28.

“We are disappointed with today’s outcome, as we believe in the strength of the data we presented for rociletinib,” said Patrick Mahaffy, Clovis’ president and chief executive. “We will work with the FDA to evaluate the best path forward as it continues to review our application.”