UPDATED: Takeda sways FDA panel with its bowel disease drug despite viral risks

After a touch-and-go review from FDA staff, Takeda's inflammatory bowel disease drug had an easier time convincing an agency advisory panel of its safety and efficacy. But while an FDA committee took little issue with vedolizumab's risk profile, Takeda may have a hard time convincing regulators to make its treatment an early option for patients.

The panel was nearly unanimous that the benefits of Takeda's drug outweighed its risks in Crohn's disease and ulcerative colitis, the two most common inflammatory bowel diseases. However, Takeda still might not get indications it wants: The group voted 20-0 with one abstention that vedolizumab had proven itself as a maintenance therapy for Crohn's but split 12-9 over whether the drug was effective as an induction therapy for the disease.

The FDA's efficacy concerns stem from some up-and-down data in vedolizumab's pivotal trials. While the drug hit its primary and secondary goals for ulcerative colitis, it initially failed a study on Crohn's. In a do-over, the drug beat out placebo but never managed to nail its secondary endpoints.

On the bright side for Takeda, the panel was largely unmoved by concerns about vedolizumab's ties to a deadly brain infection. In FDA documents posted ahead of the meeting, staff members raised questions over the risk of progressive multifocal leukoencephalopathy (PML), even though not one patient in vedolizumab's clinical trials came down with the virus. The panel, however, voted 21-0-0 that the company had adequately characterized the drug's risks of PML and agreed almost unanimously that vedolizumab was safe in tandem with immunosuppressants.

The FDA is not required to follow the recommendations of its panels, though it generally does. The agency is scheduled to render a final ruling on the ulcerative colitis application by Feb. 18 and decide on Crohn's by June 18.

"People with ulcerative colitis or Crohn's disease are in need of additional treatment options, as many patients lose response to currently available treatments," Takeda Vice President Asit Parikh said in a statement. "Vedolizumab was designed to treat inflammation in the GI tract, and if approved, may offer an additional option for patients suffering from ulcerative colitis or Crohn's disease."

Vedolizumab is a leftover from Takeda's $8.8 billion buyout of Millennium, and, as one of 6 therapies the company expects to get approved next year, it's a cornerstone of the Japanese drugmaker's near-term strategy. The drug is targeted toward patients for whom TNF-alpha blockers like Remicade and Humira don't work, and Takeda is counting on its leukocyte-targeting therapy to snag a sizable share of the multibillion-dollar market for inflammatory bowel disease treatments.

- read Takeda's release