Cemdisiran Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Cemdisiran comprises 2 peer-reviewed publications across 2 journals, 0 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Myasthenia Gravis, and Hemoglobinuria, Paroxysmal. Most recent publication: Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial., Lancet, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.
    Vu T, Habib AA, Jacob S, et al. · Lancet · 2026
    PubMed: PMID 42030965 · NCT05070858 (NIMBLE) · Myasthenia Gravis
  2. Safety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria.
    Jang JH, Wong RSM, Hartford C, et al. · EJHaem · 2025
    PubMed: PMID 40708708 · NCT04811716 · Hemoglobinuria, Paroxysmal

Publications by year

2025–2026: 2 publications.

2025
1
2026
1

Publications by indication

Myasthenia Gravis (1)

  • Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.
    Lancet · 2026 · PMID 42030965 · NCT05070858

Hemoglobinuria, Paroxysmal (1)

  • Safety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria.
    EJHaem · 2025 · PMID 40708708 · NCT04811716

Publications by journal

  • Lancet1 paper
  • EJHaem1 paper

All Cemdisiran publications (2)

2026 (1 paper)

  1. Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.
    Vu T, Habib AA, Jacob S, et al. · Lancet · 2026 · Derived
    PubMed: PMID 42030965 · NCT05070858 (NIMBLE) · Myasthenia Gravis

2025 (1 paper)

  1. Safety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria.
    Jang JH, Wong RSM, Hartford C, et al. · EJHaem · 2025 · Derived
    PubMed: PMID 40708708 · NCT04811716 · Hemoglobinuria, Paroxysmal

Sources and methodology

  • Publications: ctgov.study_references (PubMed PMIDs auto-attached by ClinicalTrials.gov to each trial), reference types RESULT, DERIVED, and BACKGROUND.
  • Per-arm outcome values: ctgov.outcome_measurements joined to ctgov.design_outcomes where outcome_type = 'PRIMARY', restricted to phase PHASE3 and PHASE2/PHASE3.
  • Publication metadata (title, journal, year, authors): PubMed E-utilities efetch.fcgi
  • Data freshness: Thu, 14 May 2026 22:07:07 GMT.
This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).