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: None *
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
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Belgium, Brazil, Canada, China, Colombia, Czech Republic, Denmark, France, Georgia, Germany, Greece, Hong Kong, 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
Active Clinical Trial Count: 17
Highest Development Phases
Phase 3: Geographic Atrophy|Macular Degeneration|Muscle Weakness|Myasthenia Gravis|Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria
Phase 1: Healthy Volunteers|Myositis, Inclusion Body
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| ACCESS-EXT | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2029-02-05 |
|
| jRCT2031230013 | P3 |
Not yet recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2028-08-02 |
|
| R3918-MG-2018 | P3 |
Unknown Status |
Myasthenia Gravis |
2028-03-23 |
|
| ACCESS-EXTENSION | P3 |
Active, not recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2028-02-16 |