Drugs / Cemdisiran
Cemdisiran
also known as
ALN-CC5 · AD-62643 · AD-62643 FREE ACID · ALN-62643 · ALN-62643 FREE ACID · Cemdisiran sodium
Small interfering RNA (siRNA) targets C5
Trials 15 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT06479863 | Aug 2024 → May 2027 expected | idiopathic inflammatory myopathy, inclusion body myositis | Austin Neuromuscular Center | Recruiting | No outcome recorded |
| Phase 12 trials | ||||||
| Phase 1 | NCT04940364 | Aug 2021 → Jun 2022 | — | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT04601844 | Nov 2020 → Jul 2021 | — | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT04811716 | Jul 2021 → Oct 2022 | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT04888507 | Jul 2021 → May 2022 | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03999840 | Jan 2021 → Jan 2024 | atypical hemolytic-uremic syndrome | Mario Negri Institute for Pharmacological Research | Withdrawn | No outcome recorded |
| Phase 2 | NCT03841448 | Sep 2019 → Mar 2022 | IgA glomerulonephritis | Alnylam Pharmaceuticals | Terminated | No outcome recorded |
| Phase 2 | NCT03303313 | Sep 2017 → Sep 2018 | atypical hemolytic-uremic syndrome | Alnylam Pharmaceuticals | Withdrawn | No outcome recorded |
| Phase 36 trials | ||||||
| Phase 3 | NCT07154745 | Jun 2026 → Jan 2030 expected | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT06541704 | Oct 2024 → Nov 2027 expected | dry age related macular degeneration | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05744921 | Mar 2023 → Feb 2029 expected | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05131204 | Oct 2022 → Jul 2023 | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Terminated | No outcome recorded |
| Phase 3 | NCT05133531 | Aug 2022 → Oct 2026 expected | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05070858 | Dec 2021 → Jul 2025 overdue | generalized myasthenia gravis | Regeneron Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT06028594 | — | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | No longer available | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02352493 Comparator | Jan 2015 → Apr 2016 | paroxysmal nocturnal hemoglobinuria | Alnylam Pharmaceuticals | Completed | No outcome recorded |
Press releases naming this drug 17 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-06-22 | Regeneron Pharmaceuticals | Cemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG) regeneron.com ↗ |
| 2026-04-21 | Regeneron Pharmaceuticals | Cemdisiran, Dosed Subcutaneously Every 12 Weeks, Demonstrates Rapid, Deep and Sustained Disease Control in Generalized Myasthenia Gravis (gMG) Phase 3 Trial regeneron.com ↗ |
| 2024-12-07 | Regeneron Pharmaceuticals | Novel Combination of Pozelimab and Cemdisiran (Poze-Cemdi) Achieved Greater Control of Intravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Compared to Ravulizumab regeneron.com ↗ |
| 2022-08-29 | Alnylam Pharmaceuticals | Alnylam Reports Positive Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy alnylam.com ↗ |
| 2022-06-09 | Alnylam Pharmaceuticals | Alnylam Reports Positive Topline Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy alnylam.com ↗ |
| 2017-09-26 | Alnylam Pharmaceuticals | Alnylam Initiates Phase 2 Clinical Study of Cemdisiran (ALN-CC5) in Patients with Atypical Hemolytic-Uremic Syndrome (aHUS) alnylam.com ↗ |
| 2016-12-05 | Alnylam Pharmaceuticals | Alnylam Presents Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) alnylam.com ↗ |
| 2016-06-11 | Alnylam Pharmaceuticals | Alnylam Reports Initial ALN-CC5 Results in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) from Ongoing Phase 1/2 Study alnylam.com ↗ |
| 2016-06-03 | Alnylam Pharmaceuticals | Alnylam to Webcast Conference Call Discussing ALN-CC5 Phase 1/2 Data alnylam.com ↗ |
| 2015-12-17 | Alnylam Pharmaceuticals | Alnylam Initiates Dosing in PNH Patients in Phase 1/2 Clinical Trial for ALN-CC5 alnylam.com ↗ |
| 2015-12-06 | Alnylam Pharmaceuticals | Alnylam Reports that ALN-CC5 Achieves Up to 99 Percent Knockdown of Serum C5 and Up to 98 Percent Inhibition of Serum Hemolytic Activity with Durability Supportive of Once Monthly and Possibly Once Quarterly Subcutaneous Dose Regimen alnylam.com ↗ |
| 2015-06-12 | Alnylam Pharmaceuticals | Alnylam Reports Positive Initial Clinical Results for ALN-CC5, an Investigational RNAi Therapeutic Targeting Complement Component C5 for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
| 2015-02-02 | Alnylam Pharmaceuticals | Alnylam Initiates Phase 1/2 Clinical Trial for ALN-CC5, a Subcutaneously Administered RNAi Therapeutic Targeting Complement Component C5 in Development for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
| 2014-12-07 | Alnylam Pharmaceuticals | Alnylam Files Clinical Trial Application (CTA) for ALN-CC5, an RNAi Therapeutic Targeting Complement C5 in Development for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
| 2014-09-16 | Alnylam Pharmaceuticals | Alnylam Presents New Pre-Clinical Data with ALN-CC5, a Subcutaneously Administered RNAi Therapeutic Targeting Complement Component C5 in Development for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
| 2014-06-09 | Alnylam Pharmaceuticals | Alnylam Presents New Pre-Clinical Data with Development Candidate for ALN-CC5, a Subcutaneously Administered RNAi Therapeutic Targeting Complement Component C5 in Development for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
| 2013-06-20 | Alnylam Pharmaceuticals | Alnylam Scientists Present Pre-clinical Data with ALN-CC5, an RNAi Therapeutic Targeting Complement Component C5 for the Treatment of Complement-Mediated Diseases alnylam.com ↗ |
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 19 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Cemdisiran | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06479863 ↗ |
| Known as | AD-62643 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297722 ↗ |
| Known as | AD-62643 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297722 ↗ |
| Known as | ALN-62643 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297722 ↗ |
| Known as | ALN-62643 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297722 ↗ |
| Known as | ALN-CC5 | “A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome” NCT03303313 ↗ |
| Known as | Cemdisiran sodium | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297722 ↗ |
| Action | Inhibit | “suppressing liver production of complement 5 (C5) protein” PMID 33047216 ↗ Mar 2021 |
| Modality | Small interfering RNA (siRNA) | “we developed a lipid nanoparticle (LNP) formulation of small-interfering RNA against complement C5 (C5 siRNA-LNP)” PMID 35749284 ↗ Nov 2022 |
| Route | Subcutaneous | “Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection” NCT02352493 ↗ |
| Target | C5 | “suppressing liver production of complement 5 (C5) protein” PMID 33047216 ↗ Mar 2021 |