New drug for treatment-resistant forms of tuberculosis approved

Pretomanid tablets in combination with bedaquiline and linezolid has been approved by the US Food and Drug Administration (FDA). 

The drug has been approved for treating a limited and specific population of adult patients with extremely drug-resistant, treatment-intolerant or non-responsive multidrug-resistant pulmonary tuberculosis (TB). 

The FDA granted the tablets Priority Review, under which the FDA’s goal is to take action on an application within an expedited time frame and Orphan Drug Designation (ODD), which provides incentives to assist and encourage the development of drugs for rare diseases.

“The threat of antimicrobial-resistant infections is a key challenge we face as a public health agency,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD. “The bacterium that causes tuberculosis can develop resistance to the antibiotics used to treat it. Multidrug-resistant TB and extensively drug-resistant TB are public health threats due to limited treatment options.

“New treatments are important to meet national and global patient 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.” 

The safety and effectiveness of the treatment was primarily demonstrated in a study of 109 patients with extensively drug-resistant, treatment‑intolerant or non-responsive multidrug-resistant pulmonary TB (of the lungs). Of the 107 patients who were evaluated six months after the end of therapy, 95 (89 percent) were successes, which significantly exceeded the historical success rates for treatment of extensively drug-resistant TB.

Pretomanid is the second drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway, established by US Congress under the 21st Century Cures Act to advance development and approval of antibacterial and antifungal drugs to treat serious or life-threatening infections in a limited population of patients with unmet need.