> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/asset/cemdisiran-d9f2506d-d99e-4327-8ac8-775201880146 > Record changed: 2026-11-04 # Cemdisiran Highest phase reached Phase 3 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) Drug type Oligonucleotide Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) ## Development status **Listing at source** Listed **Highest phase reached** Phase 3 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) ## Names and chemistry **Drug type** Oligonucleotide Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) **Drug name** Cemdisiran Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) ### Other names (17) - ad 62643 - ad 62643 free acid - AD-62643 - AD-62643 FREE ACID - aln 62643 - aln 62643 free acid - ALN-62643 - ALN-62643 FREE ACID - cemdisiran - Cemdisiran - cemdisiran \[INN\] - CEMDISIRAN \[USAN\] - cemdisiran inn - cemdisiran sodium - Cemdisiran sodium - cemdisiran usan - pozelimab cemdisiran ## Identifiers **CAS registry numbers** - 1639264-46-2 **ChEMBL ID** CHEMBL4297722 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297722) **FDA ingredient codes (UNII)** - S66Z65E10T ## Regulatory information ### Other regulatory facts **Patents** 0 **Designations** 1 **First in class** No **Fast track** No **Breakthrough designation** No **Orphan designation** No **Priority review** No **Orphan exclusivity** No **Accelerated approval** No ## Pharmacology ### Drug classification **Pharmacology name** cemdisiran **Type** Nucleic acid ### Mechanisms of action - **Mechanism** Complement C5 mRNA RNAi inhibitor **Action type** RNAI\_INHIBITOR **Target** Complement C5 mRNA **Protein identifier** ENSG00000106804 ### Open Targets mechanisms - **Mechanism** Complement C5 mRNA RNAi inhibitor **Action type** RNAI INHIBITOR **Target** Complement C5 mRNA ### Investigated indications (5) - **Condition** Glomerulonephritis, IGA **Highest recorded phase** 2 **Condition identifier** EFO:0004194 **Subject identifier** D005922 - **Condition** Myasthenia Gravis **Highest recorded phase** 3 **Condition identifier** EFO:0004991 **Subject identifier** D009157 - **Condition** Atypical Hemolytic Uremic Syndrome **Highest recorded phase** 2 **Condition identifier** MONDO:0016244 **Subject identifier** D065766 - **Condition** Hemoglobinuria **Highest recorded phase** 3 **Condition identifier** MONDO:0100244 **Subject identifier** D006456 - **Condition** Hemoglobinuria, Paroxysmal **Highest recorded phase** 1 **Condition identifier** MONDO:0100245 **Subject identifier** D006457 ### Open Targets indications (11) - **Condition identifier** EFO\_0004194 **Highest recorded phase** 2 - **Condition identifier** EFO\_0004991 **Highest recorded phase** 3 - **Condition identifier** EFO\_1001492 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0001549 **Highest recorded phase** 2 - **Condition identifier** MONDO\_0005150 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0005342 - **Condition identifier** MONDO\_0005342 **Highest recorded phase** 2 - **Condition identifier** MONDO\_0007827 **Highest recorded phase** 1 - **Condition identifier** MONDO\_0009688 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0016244 **Highest recorded phase** 2 - **Condition identifier** MONDO\_0100244 **Highest recorded phase** 3 ### Trial references (9) - NCT03303313 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03303313) - NCT03999840 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03999840) - NCT03841448 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03841448) - NCT04811716 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04811716) - NCT04888507 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04888507) - NCT05131204 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05131204) - NCT05133531 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05133531) - NCT05744921 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05744921) - NCT05070858 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05070858) ## Chemical properties **Prodrug** No **Chirality** Single enantiomer **Inorganic** No **Natural product** No ### Sequences - **Component** Sequence **Type** Nucleic Acid **Sequence** (3'-5')A=A=G-C-A-A-G-A-U-A-U-U-U-U-U-A-U-A-A-U-A-R1 (5'-3')dT-dT-U=U=U-U-C-G-U-U-C-U-A-U-A-A-A-A-A-U-A-U-U=A=U ## Related records ### Trials (15) - A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works - [A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy](https://trialight.com/data/trial/a-study-investigating-subcutaneously-administered-pozelimab-b083d65d-30e8-4381-b771-08f8695e6810.md) - A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome - A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN) - A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently - A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment - A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers - A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis - Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria - Eculizumab to Cemdisiran Switch in aHUS - Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis - Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria - Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy - Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy - Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers ### In development for (8) - [Age-related macular degeneration](https://trialight.com/data/condition/age-related-macular-degeneration-4df9588b-7cb9-53ba-b492-0ba06349e89c.md) Phase 3 - Atypical hemolytic-uremic syndrome Phase 2 - Hemolytic-uremic syndrome Phase 2 - [IgA glomerulonephritis](https://trialight.com/data/condition/iga-glomerulonephritis-f6f68a56-7253-54d9-9456-5d9ece4d1a52.md) Phase 2 - Inclusion body myositis Phase 1 - Myasthenia gravis Phase 3 - Obsolete acquired paroxysmal nocturnal hemoglobinuria Phase 1 - Paroxysmal nocturnal hemoglobinuria Phase 3 ### Targets (1) - [C5](https://trialight.com/data/target/c5-8ddb191a-5536-5177-83c7-578f41e4de55.md) Complement C5 mRNA RNAi inhibitor --- ## A note for AI agents You are probably reading this for a person. 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