As a deadline looms today for the FDA to grant accelerated approval to a new, first-in-class medication for treating tuberculosis, a watchdog group is urging the agency not do so, because clinical trials demonstrated an “alarming increased risk of death.” The drug, which is called Sirturo, or bedaquiline, was developed by Johnson & Johnson. In a recent letter to the FDA, Public Citizen says the FDA should not confer accelerated approval – which is granted to meds to treat serious diseases and fill an unmet medical need based on a surrogate endpoint – because patents taking the drug in addition to standard TB treatment during a Phase II trial were five times likelier to die than those who took a placebo. “It is not possible to conclude that a surrogate endpoint is “reasonably likely” to predict benefit for patients — as required by FDA regulations — when the same clinical trial of a drug using that surrogate endpoint shows a significant increase in mortality, regardless of the drug’s effects on the surrogate marker,” the watchdog writes to the FDA. “The only reasonable conclusion in such cases is that the selected surrogate endpoint is, in fact, not a valid or useful marker of clinical benefit; its surrogate ‘benefit’ is trumped by increases in the adverse clinical outcome — death — nullifying the use of the favorable surrogate marker as a means of granting accelerated approval.” Public Citizen, in fact, goes on to say that the NDA, or New Drug Application, for bedaquiline should never have been brought before an FDA advisory committee for consideration. Late last month, however, the Anti-Infective Drugs Advisory Committee voted unanimously to recommend that the FDA grant accelerated approval status.
