lumacaftor
Phase 2- Indications
- Heart failure
Company intelligence / Profile preview
Completed Phase 1 bioequivalence study of lumacaftor; top-line results expected in mid/late 2024. Preparing for a Phase 2 proof-of-concept trial to test lumacaftor’s effect on cerebral blood flow autoregulation and cognitive decline in heart failure patients. Received Orphan Drug Designation from EMA and FDA for SAH indication using CFTR-correctors.
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