SILVER SPRING, Md. -- The FDA has approved bedaquiline (Sirturo) for treatment of multidrug-resistant tuberculosis as part of combination therapy, the agency announced Monday.
The decision follows a narrow 11-7 vote in favor of approval by the FDA's Anti-Infective Drugs Advisory Committee last month. Its approval was opposed by the consumer advocacy group Public Citizen, which pointed to an apparent increased risk of death associated with bedaquiline in the drug's pivotal trials.
In announcing the approval, the FDA acknowledged the risk, and indicated that it would be noted in a boxed warning on the drug's label. The warning will also highlight a risk of QT prolongation that can lead to potentially fatal cardiac arrhythmias.
Nine patients taking bedaquiline during clinical trials died, compared with two assigned to placebo.
Efficacy was demonstrated in two phase II trials. In one, with patients randomized to bedaquiline or placebo in combination with standard anti-TB drugs, the median time to clearance of Mycobacterium tuberculosis from sputum was 83 days with the active drug versus 125 days with placebo.
In the second trial, median time to sputum culture conversion was 57 days with bedaquiline.
The FDA approved the drug under its accelerated approval program, permitting the agency to use this surrogate endpoint as the main basis for its decision. Bedaquiline was also designated as an orphan drug.
"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, MD, MPH, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research, in a statement.
"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."