Janssen submits bedaquiline MAA with EMA for treatment of multi-drug resistant tuberculosis

Janssen-Cilag International NV (Janssen) today announced it has submitted a Marketing Authorisation Application to the European Medicines Agency (EMA) seeking conditional 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, bedaquiline (Ba-DA-qui-lin) could be one of the first drugs with a new mechanism of action for tuberculosis (TB) in more than 40 years and one of the first ever to be specifically indicated for MDR-TB.

"MDR-TB is a growing threat to public health and it presents a significant new treatment challenge in controlling this serious and deadly disease," said Wim Parys, M.D., Head of the Infectious Diseases therapeutic area at Janssen. "This filing 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 MDR-TB."

The discovery of bedaquiline (formerly R207910) and its unique mechanism of action were announced in a Science article by scientists at Janssen.  The antibiotic kills the bacterium that causes tuberculosis (Mycobacterium tuberculosis, M.tb) by targeting adenosine triphosphate (ATP) synthase, an enzyme that is essential 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, randomised 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, TB is the second most common cause of adult deaths worldwide and kills approximately 1.4 million people per year.The World Health Organization (WHO) estimates approximately one-third of the world's population is infected with M.tb. MDR-TB is characterised by resistance to at least two of the most powerful medicines in today's standard, four-drug regimen for drug-susceptible TB.  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. Although the European region comprises a relatively small percentage of global TB cases, the European region as defined by the World Health Organisation (WHO) accounts for a large percentage of the burden of MDR-TB.

Janssen also submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the United States for bedaquiline in June 2012, under priority review status.