Regulators in the USA have granted accelerated approval of the first new tuberculosis treatment in 40 years, with the thumbs up to Janssen’s Sirturo.
The drug, which will treat pulmonary multi-drug resistant tuberculosis as part of combination therapy in adults, was granted accelerated approval by the US Food and Drug Administration based on the surrogate endpoint of time to sputum culture conversion from two Phase II trials.
“Sirturo (bedaquiline) was first discovered in our laboratories more than a decade ago and it is gratifying to see our discovery and development lead to the accelerated approval of the first TB therapy in 40 years with a new mechanism of action,” said Paul Stoffels, chief scientific officer and worldwide chairman, pharmaceuticals, at Johnson & Johnson.
“The accelerated approval of Sirturo is a significant step in the fight against multi-drug resistant TB, which is a more difficult to treat form of TB and is on the rise in many areas worldwide,” he said.
Sirturo works by inhibiting mycobacterial ATP synthase, which is an essential enzyme for generating energy in Mycobacterium tuberculosis.
Multi-drug resistant TB, where the disease is resistant to two of the four standard treatments, is considered an orphan disease in the USA, with 98 reported patients in 2011, while 10,500 contracted the non-resistant version. However, the World Health Organization estimates that more than two million people worldwide will develop MDR-TB between 2011 and 2015.
Treatment is complex and requires up to two years of treatment with companion drugs in accordance with national TB guidelines and local MDR-TB treatment practice. The FDA approval should fast track approval of the drug in areas of the world where multi-drug resistant TB is more prevalent, such as China and India.
According to the trial results, Sirturo-treated patients had a decreased time to culture conversion and improved culture conversion rates compared with the placebo. A Phase III trial is planned for the first quarter of 2013 to study the drug for nine months compared with a placebo.
Meanwhile, the FDA has finally approved Bristol-Myers Squibb and Pfizer’s blood thinner Eliquis (apixaban) after delaying a decision twice, citing the need for more data. The drug has specifically been approved for reducing the risk of stroke and blood clots in patients with non-valvular atrial fibrillation, but it carries the warning to not be used in patients with artificial heart valves or defective heart valves.
Eliquis will compete with Johnson & Johnson’s and Bayer’s Xarelto and Boehringer Ingelheim’s Pradaxa as an alternative to warfarin.