Nonprofit Wins FDA Approval for Tuberculosis DrugAt the end of the summer, the TB Alliance announced the approval of a new drug to treat some of the most drug-resistant forms of tuberculosis (TB). This is the first time the US Food and Drug Administration (FDA) has approved a TB drug developed by a not-for-profit entity. The TB Alliance is dedicated to the discovery, development, and delivery of better, faster-acting and affordable tuberculosis drugs.
The new drug, pretomanid, works in a combination regimen with Johnson and Johnson's bedaquiline and linezolid for patients with extensively drug-resistant TB (XDR-TB) or treatment-intolerant/non-responsive multidrug-resistant TB (MDR-TB)1,2.
About TBTB is a global disease found in every country in the world. It is the leading cause of death due to infectious disease worldwide. TB is caused by the bacterium Mycobacterium tuberculosis and most commonly affects the lungs, but can also infect other organs, bones, and lymph nodes3. In 2017, 10 million people fell ill from active TB and 1.6 million died. It is an airborne disease that can be transmitted through coughing or sneezing. There are more than half a million cases of MDR-TB annually, with about 6% of those cases being XDR-TB. Current WHO figures report that 202 countries have reported cases of XDR-TB. Drug-resistant forms of TB currently accounts for close to 1 in 3 deaths due to antimicrobial resistance annually4.
The TB AllianceThe TB Alliance is a not-for-profit organization dedicated to finding faster-acting and affordable drug regimens to fight TB. Through innovative science and with partners around the globe, this organization aims to ensure equitable access to faster, better TB cures that will advance global health and prosperity. Learn more about the TB Alliance and its mission on their website.
FDA Approves New Treatment for Highly Drug-Resistant Forms of Tuberculosis
“FDA approval of this treatment represents a victory for the people suffering from these highly drug-resistant forms of the world's deadliest infectious disease. The associated novel regimen will hopefully provide a shorter, more easily manageable and highly efficacious treatment for those in need.”The 3-drug course is effective in 3 months for treatment-intolerant/non-responsive MDR-TB where treatment has been lengthy and complex. Most patients suffering from TB are prescribed a combination of up to eight antibiotics, some involving daily injections for 18 months or longer. The TB Alliance notes "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."
–Mel Spigelman, MD, President and CEO of the TB Alliance
Pretomanid is only the third new anti-TB drug approved for use by the FDA in more than 40 years, as well as the first developed and registered by a not-for-profit organization. Pretomanid will be available in the US by the end of this year. In addition to the US FDA, the TB Alliance has submitted pretomanid for review by the European Medicines Agency and has provided data to the World Health Organization (WHO) for consideration of inclusion in treatment guidelines for highly drug-resistant TB.
In clinical trials for XDR-TB, almost 90 percent of patients receiving the medication recovered after six months of treatment — almost three times the success rate of current treatment options, which involve taking antibiotic cocktails for up to two years. The TB Alliance has licensed the therapy to Pennsylvania-based pharma company Mylan NV to manufacture and sell pretomanid.
Mouse models used in TB ResearchMouse strains used to model active TB infection include the C57BL/6 and BALB/c. As standard or genetically engineered models do not fully reflect the human immune system, researchers are now turning to humanized mice for TB research. Super immunocompromised mouse models such as the CIEA NOG mouse® can be reconstituted with human hematopoietic cells and used in subsequent studies.
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