FDA panel backs accelerated approval of Janssen's bedaquiline for MDR-TB

Janssen Research & Development, a unit of US health care major Johnson & Johnson (NYSE: JNJ), says that, in a unanimous (18-0) vote yesterday, a US Food and Drug Administration advisory committee agreed that the efficacy findings for its investigational drug bedaquiline (proposed trade name Sirturo) support the proposed indication for the treatment of pulmonary multi-drug resistant tuberculosis (MDR-TB) as part of combination therapy in adults.

The committee also agreed in a vote of 11-seven that the safety findings supported the proposed indication. The advisory panel backing was widely expected given the positive briefing papers issued earlier this week (The Pharma Letter November 27). However, panelists were divided as to whether bedaquiline played a role in deaths seen in clinical studies. Some panel members said they could support the drug's approval but voted against the safety question because it did not meet the "substantial evidence" standard. Some committee members expressed concern about that "mortality imbalance," as well as elevated liver enzymes - a potential sign of liver toxicity - among patients taking the drug.

Analysts at Cowen and Co, quoted by the Chicago Tribune, have forecast peak annual sales of $300 million for bedaquiline, which would make it a fairly modest product for the diversified health care company. Representatives from Janssen said that, if bedaquiline is approved, the company would propose making the product available only through public health agencies in the USA to limit use to patients with drug-resistant tuberculosis, rather than illnesses that can be effectively treated with current drugs. However, the greatest usage of the drug is likely to be in developing countries, where drug resistant TB is more prevalent. In the USA, only about 100 cases of drug-resistant TB are seen each year.