Pretomanid for highly drug-resistant TB
In mid-August the FDA approved the novel compound pretomanid for the treatment of XDR-TB and the treatment of MDR-TB for patients intolerant of, or not responding to, standard MDR-TB treatment.
Pretomanid was approved via the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway), which aims to fast-track approval of drugs for serious diseases in limited patient populations who currently have few options for treatment. The FDA states: “The healthcare community should understand that studies on the drug may have only answered focused questions about its safety and effectiveness in a limited population of patients who have unmet needs, including a lack of available alternative treatments”.
This approval was based on the results of the Nix-TB trial, here in South Africa, which enrolled patients with XDR-TB, treatment-intolerant MDR-TB or non-responsive MDR-TB. Ninety-five out of the first 107 patients had successful outcomes (culture-negative after 6 months) on a three-drug regimen of bedaquiline, pretomanid and linezolid (BPaL).
Pretomanid belongs to a class of drugs called nitroimidazooxazines. Adverse events reported in the Nix-TB trial included peripheral neuropathy, GIT disturbances, raised transaminases, visual impairment and weight loss.
Read the full report on the FDA site.