FDA Approves J&J Drug for Multi-Drug Resistant Tuberculosis

12/31/12Follow @cathyarnst

The Food & Drug Administration today approved the first drug to treat multi-drug resistant tuberculosis, Johnson & Johnson’s (NYSE: JNJ) bedaquiline (Sirturo), an important breakthrough in the global fight against one of the world’s deadliest diseases.

Made by J&J’s Janssen Therapeutics division, based in Titusville, NJ, bedaquiline is meant for patients who have failed to respond to all other treatments. It works by blocking an enzyme critical to the replication of M.tuberculosis bacteria, and the company said it is the first new drug in 40 years to attack TB via a new mechanism of action.

Bedaquiline won accelerated approval from the FDA despite an increased incidence of death in patients on the drug during a Phase II clinical trial—nine who received bedaquiline died, compared with two deaths in the placebo arm, out of a total of 160 people in the study. The cause of those increased deaths is unclear, but the FDA is requiring that the drug carry a boxed warning alerting physicians and patients to its associated risks.

The non-profit watchdog group Public Citizen sent a letter to the FDA  on Dec. 21 strongly opposing accelerated approval of bedaquiline because the death rate was five times higher in the treatment group during the clinical trial.

Nevertheless, in a blog post accompanying the approval FDA Commissioner Margaret Hamburg said bedaquiline “represents an important new development for some patients with particularly difficult to treat forms of tuberculosis” and called it a “welcome addition” to the drugs available for the highly communicable disease. Patients with TB must take a cocktail of four drugs for six to 12 months, and those with drug-resistant TB, which is on the rise around the world, undergo drug regimens for more than a year. If the treatment is inadequate or incomplete, patients can become resistant to two or more of the drugs in the cocktail, the definition of multi-drug resistant TB. Bedaquiline, combined with some of the other drugs in the cocktail, is a last ditch effort for those patients.

Tuberculosis is currently more prevalent globally than any time in history, according to the FDA. The Centers for Disease Control and Prevention reports that there were approximately 9 million people diagnosed with tuberculosis worldwide in 2011, and 1.4 million died of the disease. A total of 10,528 cases were reported in the U.S. According to the World Health Organization there were 310,000 cases of multi-drug resistant TB in 2011, and WHO predicts that more than two million people will develop the multi-drug resistant form between 2011 and 2015.

Xconomist Stewart Parker, CEO of the Infectious Disease Research Institute in Seattle, and Jim Connolly, president and CEO of Rockville, MD-based Aeras, wrote in September about the critical need for a vaccine against tuberculosis. “The global health research community must build on the growing momentum of TB research to produce breakthrough vaccines, drugs and diagnostic tools, particularly given the growing threat of multi-drug resistant TB. To do this, they will require increased investment in this research,” they wrote.

By posting a comment, you agree to our terms and conditions.

  • RC

    Could you please explain why this is better than a placebo? From your description it appears worse.