Nonprofit Wins FDA Approval for Tuberculosis Drug

TB Alliance, which is headquartered in New York and Pretoria, South Africa, became the first nonprofit organization to win regulatory approval from the U.S. Food and Drug Administration for a new drug aimed at treating some of the most drug-resistant forms of tuberculosis (TB).

On Wednesday, the FDA greenlit Pretomanid under the Limited Population Pathway for Antibacterial and Antifungal Drugs. Pretomanid was approved as part of a three-drug treatment regimen for people with extensively drug-resistant TB, multi-drug resistant TB or for patients who are treatment-intolerant or non-responsive. The three-drug regimen consists of bedaquiline, pretomanid and linezolid and is referred to as the BPaL regimen. The combination treatment was studied in the Nix-TB trial across three sites in South Africa.

Data from the Nix-TB trial showed that within six months of treatment, the combination therapy hit the mark in 90% of patients within six months, the nonprofit said. With Wednesday’s approval, Pretomanid became only the third new anti-TB drug in more than 40 years, as well as the first to be developed and registered by a not-for-profit organization.

Tuberculosis is the leading infectious cause of death worldwide. In 2017, 10 million people fell ill from active TB and 1.6 million died, TB Alliance said in its announcement. Tuberculosis is an airborne disease that can be spread by coughing or sneezing. Drug-resistant forms of TB currently accounts for close to one in three deaths due to antimicrobial resistance annually, the nonprofit group said.

Mel Spigelman, president and chief executive officer of TB Alliance, called the FDA approval of Pretomanid a “victory for the people suffering from these highly drug-resistant forms of the world's deadliest infectious disease.” He expressed hope that the treatment regimen will provide a shorter and more manageable treatment for those patients in need.

­“Until very recently, people infected with highly drug-resistant TB had poor treatment options and a poor prognosis,” Francesca Conradie, principal investigator of the Nix-TB trial, said in a statement. “This new regimen provides hope with nine out of 10 patients achieving culture negative status at six months post-treatment with this short, all-oral regimen.”

Pretomanid is a new chemical entity and a member of a class of compounds known as nitroimidazooxazines. TB Alliance acquired the developmental rights to the compound in 2002. It was granted Priority Review, Qualified Infectious Disease Product, and Orphan Drug status.

The LPAD pathway that was used for the approval of Pretomanid was established by FDA as a tool to encourage further development of antibacterial and antifungal drugs to treat serious, life-threatening infections that affect a limited population of patients with unmet needs.

Pretomanid is expected to be available in the United States by the end of this year. In addition to the U.S. FDA, TB Alliance has submitted Pretomanid as part of the BPaL regimen for review by the European Medicines Agency and has provided data to the World Health Organization for consideration of inclusion in treatment guidelines for highly drug-resistant TB.