Merck ($MRK) and Ariad Pharmaceuticals ($ARIA) saw their hopes for approval of their targeted cancer drug ridaforolimus dim after an FDA panel voted not to recommend approval of the therapy for sarcomas. U.S. regulators have the final say on the companies' bid for approval and will likely decide its fate before a June 5 deadline.
Thirteen experts on the panel voted "no" to the question of approval, with only one "yes" vote. Many analysts expected the panel to deny Merck and Ariad, especially after an FDA staff briefing questioned whether the experimental treatment's marginal benefits justified the health hazards that could hit patients while taking it. FDA staff noted that the drug kept patients' cancer from advancing for 3.1 weeks longer than a placebo, Bloomberg reported.
If Whitehouse Station, NJ-based Merck fails to sail to an FDA approval with ridaforolimus, the blow won't sink the pharma giant. Cambridge, MA-based Ariad, which has no products on the market, and its investors have banked heavily on the success of the second-most advanced drug in its pipeline, ponatinib, which has yielded promising data as a targeted treatment for chronic myeloid leukemia. And Ariad has already reaped nearly $200 million in payments from Merck related to ridaforolimus, Xconomy reported. Those funds have helped back development of ponatinib and other cancer programs in its pipeline.
Merck announced last night that it would keep pursuing approval for ridaforolimus, a small molecule mTOR inhibitor, for soft-tissue or bone sarcomas after the tough FDA panel vote.
"Merck remains confident in the potential of the investigational agent ridaforolimus for an indication where patients have limited options," Dr. Eric Rubin, vice president of Merck's clinical oncology unit, said. "We remain committed to bringing forward this promising therapy for patients with metastatic sarcoma and look forward to further discussions with the FDA regarding this application."
In related the news, the same FDA panel that shot down ridaforolimus backed approval of GlaxoSmithKline's ($GSK) Votrient (previously approved for kidney cancer) for treating sarcomas in an 11-2 vote, Bloomberg reported. The FDA will take the panel's vote into account in making its decision about approval of the drug for combating sarcomas, with a decision expected by a deadline of May 6.
- here's Merck's release
- see Bloomberg's article
- and Xconomy's report
FDA panel backs GSK's Votrient for soft-tissue sarcoma
FDA rejects Merck's new combo cholesterol drug
Ariad's ponatinib takes center stage with promising pivotal leukemia data