Cemdisiran clinical trials
Cemdisiran is named as an intervention in 4 current clinical trials in our Spain, Italy, Poland, Canada, UK, Turkey, Brazil, South Korea, India, China, Mexico, Japan, Taiwan, USA, Germany, France, Singapore, Belgium, Denmark, Austria and Australia registers. 3 are recruiting patients now. All registered phases are Phase 3. The lead sponsor is Regeneron Pharmaceuticals. The most-studied condition is Paroxysmal Nocturnal Hemoglobinuria. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Cemdisiran is approved
All regulators →- 1 EU orphan designation, most recently for treatment of primary IgA nephropathy (20 Aug 2021, positive)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
How Cemdisiran works
All drug targets →- Complement C5 mRNA RNAi inhibitor Rnai inhibitorTarget: C5 complement C5ChEMBL only
- Age-related macular degenerationPhase 3
- Atrophic macular degenerationPhase 3
- Myasthenia gravisPhase 3
- Paroxysmal nocturnal hemoglobinuriaPhase 3
- IgA glomerulonephritisPhase 2
- Atypical hemolytic-uremic syndromePhase 2
- Inclusion body myositisEarly phase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4297722, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 14708, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
Patient leaflets for Cemdisiran
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Trials by status
Trials by phase
Clinical trials of Cemdisiran
4 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Cemdisiran in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Cemdisiran. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria with Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
2024-519709-37-00 · CTIS ↗ · Hemic and Lymphatic Diseases
|
Phase 3 | Authorised | Regeneron Pharmaceuticals Inc | Poland, Italy, Spain |
Conditions studied with Cemdisiran
4 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Regeneron Pharmaceuticals | 4 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Cemdisiran trials
How many clinical trials are testing Cemdisiran?
4 current trials in our Spain, Italy, Poland, Canada, UK, Turkey, Brazil, South Korea, India, China, Mexico, Japan, Taiwan, USA, Germany, France, Singapore, Belgium, Denmark, Austria and Australia registers name Cemdisiran (also registered as ALN-CC5) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Cemdisiran trials recruiting patients?
Yes. 3 of the 4 trials are recruiting: 3 with sites in Spain, 3 with sites in Italy, 3 with sites in Poland, 3 with sites in Canada, 2 with sites in UK, 2 with sites in Turkey, 2 with sites in Brazil, 2 with sites in South Korea, 1 with sites in India, 1 with sites in China, 2 with sites in Mexico, 1 with sites in Japan, 1 with sites in Taiwan, 1 with sites in USA, 1 with sites in Germany, 1 with sites in France, 1 with sites in Singapore, 1 with sites in Austria and 1 with sites in Australia. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Cemdisiran clinical trials?
The lead sponsor is Regeneron Pharmaceuticals. The number in brackets is how many trials each organisation leads.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Cemdisiran works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Cemdisiran works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Cemdisiran (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.