Janssen seeks accelerated FDA approval for bedaquiline to treat MDR-TB

Janssen Research & Development, LLC (Janssen) today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for the use of the investigational drug bedaquiline (TMC207) as an oral treatment, to be used as part of combination therapy for pulmonary, multi-drug resistant tuberculosis (MDR-TB) in adults. If approved by the FDA, bedaquiline would be the first drug with a new mechanism of action for TB in more than 40 years and the first and only one specifically indicated for MDR-TB.

"The emergence of multi-drug resistant strains of TB is a growing problem that impacts people around the world and is posing a significant new treatment challenge in controlling this serious and deadly disease," said Paul Stoffels, Worldwide Chairman, Pharmaceuticals, Johnson & Johnson. "Although tuberculosis kills approximately 1.4 million people per year and current therapies do not provide adequate control of resistant strains, there have been no new treatment options to treat TB in the last 40 years. We believe the NDA submission for bedaquiline is an exciting milestone in the development of new TB drugs."

Bedaquiline was discovered by scientists at Janssen, a Johnson & Johnson company. Its unique mechanism of action targets adenosine triphosphate (ATP) synthase, which Mycobacterium tuberculosis (M.tb)--the bacterium that causes tuberculosis--requires to generate its energy.

The regulatory submission 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 evaluated the safety and efficacy of bedaquiline versus placebo in the treatment of patients with pulmonary MDR-TB in combination with a background regimen. 

Among infectious diseases, tuberculosis is the second most common cause of adult deaths worldwide. The World Health Organization (WHO) estimates approximately one-third of the world's population is infected with M.tb and the disease is responsible for nearly 3,800 deaths per day, worldwide. MDR-TB, which is characterized by resistance to at least two of the most powerful medicines in today's standard, four-drug regimen for drug-susceptible TB, is of particular concern given the alarming increase in antibacterial resistance throughout the world and the difficulties in treating it. In 2010, there was an estimated prevalence of 650,000 cases of MDR-TB, and in 2008 it was estimated there were 150,000 MDR-TB deaths annually. The WHO issued a Call to Action in Beijing in 2009 because of the growing public health threat it poses. In the United States, TB is an orphan disease, with approximately 100 to 130 MDR TB patients annually.

"This is a critically important milestone in the development of bedaquiline and an important step forward in the development of new treatments for TB," said Wim Parys, M.D., Head of the Infectious Diseases therapeutic area at Janssen. "It underscores our commitment to discover and develop novel medicines and solutions for serious unmet medical needs and we hope this new treatment will become an important option for patients with multi-drug resistant TB. This first submission will be followed by others in high-burden countries."