On the last day of the year the FDA came through with a rapid-fire approval of Johnson & Johnson's bedaquiline, the first new treatment for drug-resistant tuberculosis in four decades. The approval was based on mid-stage data after J&J ($JNJ) made its case that the urgent need for a new TB therapy warranted an OK ahead of Phase III results.
J&J will sell the drug as Sirturo, part of a combination therapy that will now be available as a last-ditch treatment for patients who have failed standard remedies. The FDA handed out the accelerated approval based on rules that allow the agency to green-light a therapy on efficacy data based on a surrogate endpoint.
The drug is aimed right at adenosine triphosphate synthase, cutting off the energy flow Mycobacterium needs to trigger tuberculosis. J&J generated 24-week data from an open label trial as well as a randomized study in their Phase II program.
"Multi-drug resistant tuberculosis poses a serious health threat throughout the world, and Sirturo provides much-needed treatment for patients who have don't have other therapeutic options available," said Edward Cox, M.D., M.P.H, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research. "However, because the drug also carries some significant risks, doctors should make sure they use it appropriately and only in patients who don't have other treatment options."
"Sirturo was first discovered in our laboratories more than a decade ago and it is gratifying to see our discovery and development lead to the accelerated approval of the first TB therapy in 40 years with a new mechanism of action. This underscores our commitment as a company to discover, develop and responsibly deliver innovative medicines that address serious unmet medical needs," said Paul Stoffels, J&J's CSO.
The FDA used the last day of the year to add to its record roster of approvals, which has now topped the 36 approvals seen in 2004, as reported recently by the FDA.
- here's the FDA's release
- and the J&J release
Slideshow: FDA Approvals of 2012