Johnson & Johnson has filed bedaquiline with regulators in the USA hoping it will be the first drug with a new mechanism of action for tuberculosis in more than 40 years.
Specifically, J&J's Janssen unit is seeking accelerated approval from the US Food and Drug Administration for bedaquiline as an oral treatment to be used as part of combination therapy for pulmonary, multi-drug resistant TB (MDR-TB) in adults. The company said that its unique mechanism of action targets adenosine triphosphate synthase, which Mycobacterium tuberculosis (the bacterium that causes tuberculosis) requires to generate its energy.
If approved, bedaquiline also would be the first medicine specifically for treating MDR-TB, which is characterised by resistance to "at least two of the most powerful medicines in today's standard, four-drug regimen for drug-susceptible TB", J&J noted. In 2010, there were around 650,000 cases of MDR-TB; estimates have suggested 150,000 people die every year as a result of the latter.
Paul Stoffels, chairman of J&J's pharmaceuticals group, noted that the emergence of MDR-TB is "posing a significant new treatment challenge in controlling this serious and deadly disease". He noted that although TB kills approximately 1.4 million people per 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 treatment with bedaquiline versus placebo is planned to start recruiting in the fourth quarter. The study will evaluate a new regimen of seven drugs for a shorter treatment duration (nine months of treatment) than the current 18 to 24 months recommended by the World Health Organisation.
Wim Parys, head of infectious diseases 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.