FDA panel questions whether bowel disease drug increases risk of deadly brain infection

fda logo FDA panel questions whether bowel disease drug increases risk of deadly brain infectionA Food and Drug Administration (FDA) advisory panel is questioning the safety of Takeda Pharmaceutical’s inflammatory bowel disease drug candidate vedolizumab, claiming the drug could cause patients to develop a rare but deadly brain infection. The panel is set to rule on Dec. 9 whether to recommend the drug be approved for marketing in the United States.

The medication fared well in clinical trials as a treatment for ulcerative colitis and Crohn’s disease, but experts say the drug carries the risk of progressive multifocal leukoencephalopathy (PML), a life threatening brain disease that affects immune deficient patients.

No patients in clinical trials with vedolizumab developed PML, however the drug works in much the same way as Biogen Idec’s Tysabri, an anti-inflammatory medication that already carries a black box warning for PML. Takeda claims its drug is mechanistically different than Tysabri, which makes it safer, but the agency isn’t convinced.

Efficacy data for vedolizumab isn’t clear cut. The drug hit its primary and secondary endpoints for ulcerative colitis and fared well against placebo in a second trial on Crohn’s disease after failing a prior study. But it missed secondary goals as a treatment for Crohn’s. This led the FDA to question whether the drug has proven itself as a valid treatment for Crohn’s. The panel may also restrict its indications and require Takeda to conduct costly premarket safety studies to better identify the risk of PML.

Regardless of the advisory panel’s recommendation on vedolizumab, it is up to the FDA to ultimately decide the drug’s fate. Regulators are expected to make a final ruling on the medication by February 18.

Source: Fierce Biotech