Tuberculosis Drug, Bedaquiline, Submitted for FDA New Drug Application Approval

Johnson & Johnson’s Janssen Research & Development unit yesterday announced that they have filed a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bedaquiline, in the hope that it will be the first drug with a new mechanism of action for tuberculosis (TB) in over 40 years.

Specifically, Janssen is pursuing approval for the drug as an oral treatment to be used as part of combination therapy for pulmonary, multi-drug resistant tuberculosis (MDR-TB) in adults.  The organisation noted that their unique mechanism of action targets adenosine triphosphate synthase, which Mycobacterium tuberculosis (the bacterium that causes tuberculosis) requires to generate its energy.

If approved, bedaquiline would also be the first medication intended for treating multi-drug resistant tuberculosis, which is defined as having resistance to “at least two of the most powerful medicines in today’s standard, four-drug regimen for drug-susceptible TB,” Johnson and Johnson commented.

The submission to the US FDA is supported by 24-week data from the Phase II clinical development program, which includes an open-label study and a controlled, randomized trial that assessed the safety and efficacy of bedaquiline.

In 2010, there were approximately 650,000 cases of MDR-TB; and estimations have suggested that roughly 150,000 people die each year as a result of MDR-TB.

Chairman of Johnson and Johnson pharmaceuticals group, Paul Stoffels, commented that the appearance of MDR-TB is “posing a significant new treatment challenge in controlling this serious and deadly disease.”

He added that although tuberculosis kills roughly 1.4 million people each year, “current therapies do not provide adequate control of resistant strains and there have been no new treatment options to treat TB in the last 40 years.”

A Phase III trial comparing nine months of medication with bedaquiline in contrast with a placebo is scheduled to begin enrolling in the fourth quarter.  The clinical study will assess a new regimen of seven drugs for a shorter treatment length than the current 18 to 24 months recommended by the World Health Organisation (WHO).

Wim Parys, head of infectious diseases therapeutic area at Janssen noted that this first submission “will be followed by others in high-burden countries”. In the USA, TB is an orphan disease, with 100 to 130 MDR-TB patients annually.


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