FDA grants brensocatib priority review for bronchiectasis
Key takeaways:
- Brensocatib given as 25 mg performed better than placebo when assessing rate of pulmonary exacerbations and lung function changes.
- The Prescription Drug User Fee Act goal deadline is Aug. 12.
The FDA has granted priority review to brensocatib to treat patients with non-cystic fibrosis bronchiectasis, according to a manufacturer-issued press release.
If approved, the release said this will be the first treatment for this patient population.
“Right now, people with bronchiectasis have no approved treatments to prevent pulmonary exacerbations that define this disease and can lead to destruction of lung tissue, putting hundreds of thousands at risk of experiencing accelerated lung function decline,” Martina Flammer, MD, MBA, chief medical officer of Insmed, told Healio. “The FDA’s priority review of brensocatib recognizes this unmet need and is a major milestone for the entire bronchiectasis community, bringing us one step closer to potentially delivering the first-ever treatment specifically approved for bronchiectasis as soon as possible.”
The FDA based this decision, in part, on findings from the global, randomized, double-blind, placebo-controlled phase 3 ASPEN trial including 1,680 adults and 41 adolescents with non-cystic fibrosis bronchiectasis. The trial determined the impact of once-daily 10 mg brensocatib (Insmed) and 25 mg brensocatib vs. placebo on pulmonary exacerbation rate, lung function, quality of life and safety at 52 weeks.
As Healio previously reported, by the 52-week mark, patients receiving 10 mg brensocatib vs. placebo had a 21.1% reduction in the annualized rate of exacerbations (P = .0019). Researchers observed a similar significant decrease in this exacerbation rate by 19.4% when comparing patients receiving 25 mg brensocatib with those receiving placebo (P = .0046).
When assessing the change in baseline FEV1 at 52 weeks, researchers noted a significant difference between the 25 mg brensocatib group and the placebo group (38 mL; P = .0054), and this was also the case when evaluating the change in baseline FVC (75 mL; P < .0001) because the 25 mg brensocatib group experienced less loss of FVC vs. the placebo group (–12 mL vs. –87 mL).
Patients also had improved quality of life with 52-week once-daily brensocatib vs. placebo based on two assessments: the bronchiectasis exacerbation and symptom tool (BEST) score and the Quality of Life-Bronchiectasis questionnaire Respiratory Symptom Domain score.
In terms of safety, the release noted that the drug was well tolerated by patients.
The most common treatment-emergent adverse event in the brensocatib groups was COVID-19, which impacted 15.8% of patients receiving 10 mg brensocatib, 20.9% of patients receiving 25 mg brensocatib and 15.8% of patients receiving placebo.
Other adverse events that impacted the brensocatib groups more included nasopharyngitis (10 mg, 7.7% vs. 25 mg, 6.3% vs. placebo, 7.6%), cough (10 mg, 7% vs. 25 mg, 6.1% vs. placebo, 6.4%) and headache (10 mg, 6.7% vs. 25 mg, 8.5% vs. placebo, 6.9%).
The Prescription Drug User Fee Act goal deadline is Aug. 12, according to the release.