- Amikacin liposome inhalation suspension (ALIS) for up to 20 months achieved additional culture conversions without new safety signals in patients with refractory Mycobacterium avium complex (MAC) lung disease.
Why this matters
- Safety concerns previously precluded ALIS use beyond 6 months.
- Open-label extension of CONVERT (phase 3 trial comparing ALIS+guideline-based therapy [GBT] vs GBT alone for refractory MAC lung disease) evaluating prolonged ALIS+GBT.
- 163 CONVERT subjects (73 ALIS+GBT, 90 GBT-alone arm) without culture conversion by month 6 received once-daily nebulized ALIS (590 mg) for up to 12 months.
- Culture conversion defined as 3 consecutive MAC-negative monthly sputum cultures.
- Funding: Insmed Incorporated.
- By 6 and 12 months, respectively, culture conversion observed in:
- 24/90 (26.7%) and 30/90 (33.3%) prior GBT-alone patients.
- 7/73 (9.6%) and 10/73 (13.7%) prior ALIS+GBT patients.
- 96.9% had ≥1 treatment-emergent adverse event (TEAE).
- 83.3% of prior GBT-alone and 46.6% of prior ALIS+GBT patients had respiratory TEAEs.
- 4.4% of prior GBT-alone and 2.7% of prior ALIS+GBT patients had fatal TEAEs.
- Safety profile consistent with CONVERT's.
- Nonrandomized, open-label, uncontrolled study with differing durations of postconversion treatment.
- Possible participation bias among CONVERT ALIS+GBT patients who tolerated ALIS.