Approval for the oral nitroimidazooxazine antimycobacterial drug, used in an all-oral combination regimen dubbed BPaL or B-L-Pa, was overwhelmingly recommended by an FDA advisory panel in June.
The three-drug regimen with a 6-month treatment also simplifies and shortens the therapy for extensively drug resistant tuberculosis that previously might take between six and eight drugs for two years or longer.
"As the third new drug developed for TB in over half a century, and the first to be developed as part of a ready-to-use treatment regimen, pretomanid's approval represents another potent tool for tackling difficult-to-treat drug-resistant forms of TB", said Médecins Sans Frontières (MSF), in a statement.
"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".
Tuberculosis is a bacterial infection that caused about 1.6 million deaths globally in 2017, according to the World Health Organisation (WHO). XDR is resistant to all four families of antibiotics typically used to fight TB, and the existing treatment regimen is hard and can result in severe side effects-including death. This three-drug regimen was studied in a Nix-TB trial across three sites in South Africa in patients with extensively drug-resistant TB, or multi-drug resistant TB.
Everitt thinks BPaL will be a significant improvement for patients.
However, what remains the concern is the cost of the treatment.
David Dowdy, MD, PhD, a TB expert and infectious disease epidemiologist at Johns Hopkins Bloomberg School of Public Health, agrees. "The approval of this new regimen by the US FDA is just the first step".
Having a nonprofit backer could also help ensure that pretomanid is affordable, "which numerous recent new drugs for hard-to-treat diseases are not", he added. The new drug application included 18 other clinical studies evaluating pretomanid's safety and efficacy.
"That all has to be managed carefully", Everitt said.
Pretomanid was developed by TB Alliance, a not-for-profit organisation, funded by governments of Australia, Germany, the United Kingdom and the U.S., as well as philanthropic sources.
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"We welcome this approval as it shows the real-world impact of U.S. government investment in finding new cures and vaccines for the world's deadliest diseases", Global Health Technologies Coalition (GHTC) director Jamie Bay Nishi said in a statement. This approval also marks the second time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, a pathway, advanced by Congress, to spur development of drugs targeting infections that lack effective therapies.
Pretomanid is expected to be available in the United States by the end of this year.
"This newly approved regimen containing pretomanid could be a lifesaver for people with XDR-TB, but it's not time to celebrate yet", said Sharonann Lynch, HIV & TB policy advisor for MSF's Access Campaign.
"It should have a major impact in those areas", he said.