The decision comes two months after a panel of advisers to the FDA voted 14-4 in favor of the drug, pretomanid, in combination with linezolid and Johnson & Johnson’s bedaquiline for multi-drug resistant tuberculosis (MDR-TB) and extensively drug-resistant tuberculosis (XDR-TB).
TB Alliance’s oral, six-month treatment regimen stands out from current treatment options which use a cocktail of antibiotic drugs over a period of up to two years.
The non-profit has granted a license to Mylan NV in April to manufacture and sell pretomanid as part of certain regimens in high-income markets and a non-exclusive license in low-income and middle-income countries where most tuberculosis cases occur.
“If approved, we expect pretomanid to be available by the end of the year,” a Mylan spokeswoman told Reuters ahead of the FDA decision.
“We will focus on the United States and in countries where there is a high burden of extensively drug-resistant TB, the majority of which are low and middle-income markets.”
Tuberculosis is a bacterial infection that caused about 1.6 million deaths globally in 2017, according to the World Health Organization (WHO).
MDR-TB and XDR-TB are forms of tuberculosis that do not respond to first-line anti-TB drugs. XDR-TB, a more serious form of MDR-TB, is unresponsive even to the two most powerful anti-TB drugs – isoniazid and rifampicin – in addition to being resistant to certain second-line treatments.
WHO estimates that there were more than half a million global cases of rifampicin-resistant TB in 2017, 82% of which were MDR-TB cases. About 8.5% of MDR-TB cases had XDR-TB in the same year.
BPaL is likely to be used by only about a “handful” of patients in the United States every year, TB Alliance Chief Executive Officer Mel Spigelman had said ahead of the FDA decision, adding that there is limited available data to estimate this specific patient population.
In addition to the United States, TB Alliance filed for approval of pretomanid as part of the BPaL regimen in the European Union.