Insmed Incorporated has announced positive topline results from the Phase 3b ENCORE study evaluating ARIKAYCE (amikacin liposome inhalation suspension) in patients with Mycobacterium avium complex (MAC) lung disease. The trial compared once‑daily ARIKAYCE plus multidrug therapy (azithromycin 250 mg and ethambutol 15 mg/kg) with placebo plus the same multidrug regimen in patients with a newly diagnosed MAC lung infection who had not previously received antibiotics. According to the company, the study met its objectives and demonstrated that adding ARIKAYCE provided clinical benefit as part of the multidrug treatment regimen.
Insmed stated that completion of ENCORE fulfills a post‑marketing study requirement from the FDA for ARIKAYCE. Based on these findings, the company plans to submit a supplemental new drug application in the second half of 2026 to support a potential label expansion for ARIKAYCE in MAC lung disease and to seek traditional approval for the existing refractory indication in the United States. Insmed also plans to submit the ENCORE data to Japan’s Pharmaceuticals and Medical Devices Agency in the second half of 2026 to support potential label expansion there and intends to present full results at a future medical congress.
Company representatives and study investigators described the results as supporting earlier use of ARIKAYCE in the treatment course for patients with MAC lung disease. They highlighted reported improvements in Respiratory Symptom Score together with durable culture conversion as indicators that adding ARIKAYCE to multidrug therapy may lead to meaningful clinical benefits for patients with this serious and often progressive infection.
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