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
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | generalized myasthenia gravis | — | “The U.S. regulatory application for cemdisiran was submitted in the first quarter of 2026” regeneron.com ↗ “submission of a New Drug Application (NDA) to the FDA for cemdisiran” alnylam.com ↗ |
Trials 15
| 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 · 1 missed | ||||||
| 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| Jan 2021 → Jan 2024 | atypical hemolytic-uremic syndrome | Mario Negri Institute for Pharmacological Research | Withdrawn | No outcome recorded |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 | Missed primary |
| Phase 2 | NCT03303313 | Sep 2017 → Sep 2018 | atypical hemolytic-uremic syndrome | Alnylam Pharmaceuticals | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 36 trials · 1 met primary | ||||||
| Phase 3 | NCT07154745 | Jun 2026 → Jan 2030 expected | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT06541704 | Oct 2024 → Jul 2028 expected | dry age related macular degeneration | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05744921 | Mar 2023 → Oct 2028 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 Stop: Business |
| 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 | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT06028594 | — | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | No longer available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1/2 | NCT02352493 Comparator | Jan 2015 → Apr 2016 | paroxysmal nocturnal hemoglobinuria | Alnylam Pharmaceuticals | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 15
| Date | Issuer | Release |
|---|---|---|
| 2026-06-22 | Regeneron Pharmaceuticals | Regulatory Cemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG) regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have accepted the regulatory applications for cemdisiran to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. |
| 2026-04-21 | Regeneron Pharmaceuticals | Results Cemdisiran, Dosed Subcutaneously Every 12 Weeks, Demonstrates Rapid, Deep and Sustained Disease Control in Generalized Myasthenia Gravis (gMG) Phase 3 Trial regeneron.com ↗
In the Phase 3 NIMBLE trial, this first-of-its-kind investigational therapy demonstrated rapid, robust efficacy with lasting benefit through 24 weeks. |
| 2025-08-26 | Regeneron Pharmaceuticals | Results Regeneron Announces Positive Results from Phase 3 Trial in Generalized Myasthenia Gravis regeneron.com ↗ |
| 2024-12-07 | Regeneron Pharmaceuticals | Results 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 ↗
Head-to-head exploratory cohort of a Phase 3 trial showed first-in-class poze-cemdi combination treatment helped patients achieve and maintain greater disease control, as measured by lactate dehydrogenase (LDH) levels, compared to standard-of-care ravulizumab |
| 2022-08-29 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy alnylam.com ↗
Treatment with Cemdisiran Resulted in a Higher Proportion of Patients Achieving Clinically Meaningful Reductions in 24-Hour Urine Protein to Creatinine Ratio, as Compared to Patients on Placebo |
| 2022-06-09 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy alnylam.com ↗
At Week 32, treatment with cemdisiran resulted in a 37 percent mean reduction from baseline in the 24-hour urine protein to creatinine ratio relative to placebo – the primary endpoint of the study and an important prognostic marker of disease progression. |
| 2019-02-07 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2018 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Alnylam announces today that it has received regulatory approval to initiate a Phase 2 study of cemdisiran in IgA nephropathy. |
| 2016-12-05 | Alnylam Pharmaceuticals | Results Alnylam Presents Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) alnylam.com ↗
New results show that ALN-CC5-mediated knockdown of serum C5 has the potential to enable effective sparing of eculizumab in patients with PNH. |
| 2016-06-11 | Alnylam Pharmaceuticals | Results Alnylam Reports Initial ALN-CC5 Results in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) from Ongoing Phase 1/2 Study alnylam.com ↗
Initial Results Also Support Potential for ALN-CC5 to Improve Disease Control in Eculizumab Inadequate Responders |
| 2015-12-06 | Alnylam Pharmaceuticals | Results 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 ↗
New results show that ALN-CC5 achieved up to 99 percent knockdown of serum C5 and up to 98 percent inhibition of serum hemolytic activity, an assay for complement activity. |
| 2015-06-12 | Alnylam Pharmaceuticals | Results 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 ↗
Initial study results from 12 healthy volunteer subjects showed that single subcutaneous dose administration of ALN-CC5 resulted in potent, dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96%. |
| 2014-12-07 | Alnylam Pharmaceuticals | Regulatory 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 | Results 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 ↗
Alnylam Pharmaceuticals, Inc . (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today new pre-clinical results with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases. |
| 2014-06-09 | Alnylam Pharmaceuticals | Results 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 ↗
New data demonstrate that ALN-CC5 led to an up to 98.7% knockdown of serum C5 and an up to 96.8% inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration. |
| 2013-06-20 | Alnylam Pharmaceuticals | Results 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 ↗
Alnylam Pharmaceuticals , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5. |
All press releases naming this drug 19 releases
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 NCT04811716 ↗ |
| 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 |