The Janssen Pharmaceutical Companies of Johnson & Johnson announced this week that the U.S. Food and Drug Administration (FDA) has granted approval for a new pediatric formulation of SIRTURO® (bedaquiline). SIRTURO® is now indicated for use as part of combination therapy in the treatment of adult and pediatric patients (5 years and older and weighing at least 15 kg) with pulmonary multidrug-resistant tuberculosis (MDR‑TB). In the U.S., the medicine should be reserved for use when an effective treatment regimen cannot otherwise be provided.

Scanning electron micrograph of Mycobacterium tuberculosis, the bacteria that cause tuberculosis. NIAID

This indication received accelerated approval based on time to sputum culture conversion. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. SIRTURO® should not be used for the treatment of latent TB infection, extra-pulmonary or drug-sensitive TB, or for the treatment of infections caused by non-tuberculous mycobacteria. The safety and efficacy of SIRTURO® in the treatment of HIV-infected patients with MDR-TB have not been established, as clinical data are limited.

The decision marks the first regulatory approval for the pediatric formulation of SIRTURO® and is a key component of Johnson & Johnson’s global pediatric research and development (R&D) program for the medicine. The new 20 mg tablet can be administered with water for patients who are able to swallow the intact tablet and taken with food. For patients who have difficulty swallowing intact tablets, the tablet can be dispersed in water and administered. To aid with administration, the dispersed mixture in water can be further mixed with a beverage or soft food. Alternatively, the tablet can be crushed and mixed with soft food immediately prior to use and administered.

“The first-ever approval of a pediatric formulation of bedaquiline is a significant advancement for children with multidrug-resistant tuberculosis,” said Martin Fitchet, M.D., Global Head, Global Public Health, Johnson & Johnson. “TB is already an often-overlooked area in global health, and children with the disease are especially vulnerable. Modernizing pediatric treatment is a critical step toward reducing the suffering of these young patients and ending TB once and for all.”

When SIRTURO® first received accelerated approval from the U.S. FDA for use in eligible adult patients in 2012, it was the first novel TB medicine in more than 40 years. In 2019, the FDA granted approval for SIRTURO® 100 mg tablets as part of combination therapy in adolescent patients (12 to less than 18 years of age and weighing at least 30 kilograms (66 pounds)) with pulmonary MDR-TB, when an effective treatment regimen cannot otherwise be provided. Further research is ongoing in children aged two to four, and in infants younger than two years old.

TB is the world’s deadliest infectious disease, claiming approximately 1.5 million lives in 2018 alone – more than HIV and malaria combined. While TB most often affects adults in their most productive years, in 2018, an estimated 1.1 million children became ill with TB worldwide and more than 200,000 died. According to the World Health Organization, however, these are likely underestimates of the true burden of the disease in children. These grim statistics underscore the urgent need for effective pediatric TB treatments.

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“In the last 10 years, we have seen great advances in innovation for tuberculosis, especially for the hardest to treat forms,” said Ruxandra Draghia-Akli, M.D., Ph.D., Global Head, Global Public Health R&D, Janssen Research & Development, LLC. “Johnson & Johnson is proud to be driving this research and development for patients of all ages. This latest accomplishment for our bedaquiline pediatric program will provide a new tool to address MDR-TB in vulnerable populations.”

The FDA approval is supported by evidence from a single-arm, open-label, Phase 2 study that enrolled pediatric patients aged 5 to less than 12 years of age with confirmed or probable pulmonary MDR-TB infection who were treated at half the adult dose with the SIRTURO® 20mg tablet for 24 weeks in combination with a background regimen for the treatment of MDR-TB. The application for the pediatric formulation obtained priority review from the FDA.