Product Description
Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases. Cemdisiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform. (Sourced from: https://investors.alnylam.com/press-release?id=26771)
Mechanisms of Action: C5 Inhibitor
Novel Mechanism: No
Modality: Nucleic Acid
Route of Administration: Subcutaneous
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Regeneron
Company Location: TARRYTOWN NY 10591
Company CEO: Leonard S. Schleifer
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Austria, Belgium, Brazil, Canada, China, Colombia, Czech Republic, Denmark, France, Georgia, Germany, Greece, Hungary, India, Italy, Japan, Jordan, Korea, Malaysia, Mexico, Peru, Philippines, Poland, Romania, Serbia, Singapore, South Africa, South Korea, Spain, Taiwan, Thailand, Turkey, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 16
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Regeneron presented P3 Myasthenia Gravis results on 2025-08-26 for Cemdisiran
- Clinical Outcomes Reported - Regeneron announced they will present P3 Myasthenia Gravis results in 2H25 for Cemdisiran
- Clinical Outcomes Reported - Regeneron presented P3 Paroxysmal Nocturnal Hemoglobinuria results on 2024-12-07 for Cemdisiran
Highest Development Phases
Phase 3: Geographic Atrophy|Macular Degeneration|Muscle Weakness|Myasthenia Gravis|Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria
Phase 1: Myositis, Inclusion Body
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
2020-002761-33 |
- | P3 |
Active, not recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2025-11-03 |
47% |
||
NCT05070858 |
NIMBLE | P3 |
Active, not recruiting |
Myasthenia Gravis|Muscle Weakness |
2025-07-18 |
33% |
2025-08-23 |
|
NCT06541704 |
SIENNA | P3 |
Recruiting |
Geographic Atrophy|Macular Degeneration |
2027-11-29 |
99% |
2025-07-31 |
Patient Enrollment|Primary Endpoints|Treatments |
NCT06479863 |
R3918-OT-2383 | P1 |
Recruiting |
Myositis, Inclusion Body |
2027-05-30 |
83% |
2024-09-20 |
Primary Endpoints|Start Date|Treatments|Trial Status |
NCT07154745 |
R3918-PNH-2483 | P3 |
Not yet recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2031-01-25 |
2025-09-05 |
Primary Endpoints|Treatments |
|
2023-510336-36-00 |
R3918-PNH-2050 | P3 |
Recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2029-05-02 |
42% |
2025-05-02 |
Treatments |
NCT05744921 |
ACCESS-EXT | P3 |
Recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2029-02-26 |
42% |
2025-06-26 |
|
jRCT2031230013 |
jRCT2031230013 | P3 |
Not yet recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2028-08-02 |
|||
2023-508842-17-00 |
R3918-MG-2018 | P3 |
Active, not recruiting |
Myasthenia Gravis |
2028-03-23 |
33% |
2025-05-02 |
Treatments |
jRCT2071220059 |
jRCT2071220059 | P3 |
Recruiting |
Myasthenia Gravis |
2027-04-30 |
|||
2023-509657-31-00 |
R3918-PNH-2021 | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria |
2027-04-01 |
42% |
2025-05-02 |
Treatments |
NCT05133531 |
ACCESS-1 | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2027-01-28 |
42% |
2025-08-27 |
|
jRCT2031220440 |
jRCT2031220440 | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2026-09-27 |
|||
2020-005005-17 |
2020-005005-17 | P2 |
Completed |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2024-04-23 |
67% |
2022-03-13 |
Treatments |
2024-519709-37-00 |
R3918-PNH-2483 | P3 |
Not yet recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2031-02-07 |
|||
2023-509547-27-00 |
R3918-AMD-2326 | P3 |
Not yet recruiting |
Macular Degeneration|Geographic Atrophy |
2032-01-05 |
99% |
2025-05-02 |
Treatments |