Asset profile

lumacaftor

Small molecule

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Approvedregulatory verdict

Evidence outlook

Approved: Cystic Fibrosis

Regulatory approval supersedes design-level evidence grading; the trials behind approval are preserved below as the drug's evidence history.

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Competitive landscape

7 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 7 same-indication.

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DrugStageModalityMechanismEvidence signalReported efficacygraded featured trials
Lumacaftor: this drugApprovedSmall moleculeCFTR
Elexacaftor / Tezacaftor / Ivacaftor (Trikafta)ApprovedSmall molecule—
Ivacaftor (Kalydeco)ApprovedSmall molecule—
Tezacaftor / Ivacaftor (Symdeko)ApprovedSmall molecule—
Vanzacaftor / Tezacaftor / Deutivacaftor (Alyftrek)ApprovedSmall molecule—
VX-522Phase 1/2RNA / oligonucleotide—
KB407Phase 1Gene therapy / editing—
VX-828Phase 1Small molecule—
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Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.

Upcoming events

(1 total)

Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.

2027 (1)
May 2027 (1)
Phase 2Heart Failure With Reduced Ejection FractionRecruiting
Est. primary completion 2027-05 · NCT07695090 · CT.gov ↗
What to watch
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All registered studies

Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.

Phase 3 (11)
Phase 2 (7)
Phase 1 (7)
Phase 4 (3)
Early phase / not applicable (3)
Appears in other studies (1)