The Food and Drug Administration (FDA) has approved Sirturo (bedaquiline; Janssen) for use in pediatric patients aged 12 to less than 18 years and weighing at least 30kg as part of combination therapy for pulmonary multidrug-resistant tuberculosis (MDR-TB), when an effective treatment regimen cannot otherwise be provided. Previously, the antimycobacterial agent had only been approved for use in adult patients.
The approval was based on data from a single-arm, open-label, phase 2 study that evaluated the pharmacokinetics, safety and tolerability of Sirturo in combination with a background regimen in patients aged 12 to less than 18 years with confirmed or probable pulmonary MDR-TB infection (N=15). Patients received 400mg once daily for the first 2 weeks followed by 200mg 3 times per week for the following 22 weeks.
Findings from the study showed that in the subset of patients with culture positive pulmonary MDR-TB at baseline (n=8), treatment with Sirturo resulted in conversion to a negative culture at Week 24 for 75% of patients (n=6).
With regard to safety, the most common adverse reactions were arthralgia, nausea and abdominal pain; observed laboratory abnormalities were similar to those seen in adult trials.
The expanded indication is approved under 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.
“We are proud of what today’s approval means for adolescent patients in the US, and we are hopeful about the future of our pediatric R&D program,” said Paul Stoffels, MD, Vice Chair of the Executive Committee and Chief Scientific Officer, Johnson & Johnson. “Despite being a major global health issue, TB lacks the attention and resources it requires – and the issue of TB in children is even more neglected. At Johnson & Johnson, we are committed to developing solutions that address the needs of all TB patients, including the youngest and most vulnerable.”
The Company is currently conducting a phase 2 trial of Sirturo in patients younger than 12 years of age using a pediatric formulation of bedaquiline.
Sirturo is currently supplied as 100mg tablets.
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