The approval marks the second drug approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs.
The US Food and Drug Administration (FDA) has announced the approval of pretomanid tablets in combination withbedaquiline and linezolid for the treatment of highly treatment-resistant tuberculosis. The novel compound, pretomanid, was developed by the TB Alliance.
The approval was granted under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway) as a 3-drug, 6-month, all-oral regimen for individuals with extensively-drug resistant (XDR) or multidrug-resistant (MDR) tuberculosis who are treatment intolerant or non-responsive to treatment. Pretomanid is only the third new anti-TB drug approved for use by FDA in more than 40 years.
According to a statement issued by the TB Alliance the 3-drug regimen, referred to as the BPaL regimen, was studied in the Nix-TB trial across 3 sites in South Africa. The study enrolled 109 individuals with XDR-TB or treatment-intolerant or non-responsive MDR-TB.
Data from the study indicate that 95 of the first 2017 patients experienced successful outcomes after 6 months of treatment with the regimen and 6 months of follow up, exceeding historical success rates for treatment. For 2 patients, treatment was extended to 9 months.
“Until very recently, people infected with highly drug-resistant TB had poor treatment options and a poor prognosis,” said Francesca Conradie, MBBCh principal investigator of the Nix-TB trial in a statement issued by the TB Alliance. “This new regimen provides hope with 9 out of 10 patients achieving culture negative status at 6 months post-treatment with this short, all-oral regimen."
Adverse reactions from the trial included hepatotoxicity, myelosuppression, and peripheral and optic neuropathy.
Pretomanid has been evaluated in over 19 clinical trials assessing safety and efficacy and has been studied in 14 countries.
“Multidrug-resistant TB and extensively drug-resistant TB are public health threats due to limited treatment options,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD, in a statement. “New treatments are important to meet patient national and global health needs. That’s why, among our other efforts to address antimicrobial resistance, we’re focused on facilitating the development of safe and effective new treatments to give patients more options to fight life-threatening infections.”
Pretomanid is expected to be available in the United States by the end of this year.