Drugs / Vutrisiran
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Vutrisiran

Small interfering RNA (siRNA) targets TTR via inhibition

Developed for
hereditary amyloidosis · familial amyloid neuropathy
Investigated by
Alnylam Pharmaceuticals

Regulatory milestones approvals, filings & regulatory actions · 9 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Filed AH amyloidosis 4 milestones
Filed Japan (PMDA) AMVUTTRA AH amyloidosis 2025-02-13

“Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).” alnylam.com ↗

“Parallel filings for regulatory approval have also been submitted in all major regions, including Europe and Japan .” alnylam.com ↗

Filed EU (EMA) AMVUTTRA AH amyloidosis 2024-10-16

“the submission of a Type II Variation to the European Medicines Agency (EMA) for vutrisiran” alnylam.com ↗

Filed US (FDA) AMVUTTRA AH amyloidosis 2024-10-09

“the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran” alnylam.com ↗

Filed US (FDA) — AH amyloidosis 2022-04-04

“Alnylam has identified a new facility to pack and label vutrisiran and submitted an amendment to the NDA for review by the FDA” alnylam.com ↗

Filed familial amyloid neuropathy 1 milestone
Filed US (FDA) — familial amyloid neuropathy 2021-04-29

“accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary...” alnylam.com ↗

“Vutrisiran , a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis Filed a NDA with the FDA.” alnylam.com ↗

“In June 2021 , the U.S. Food and Drug Administration (FDA) accepted the Company’s New Drug Application (NDA) submission for review for vutrisiran” alnylam.com ↗

“a New Drug Application (NDA) for the approval of vutrisiran was filed with the U.S. Food and Drug Administration (FDA) in April 2021” alnylam.com ↗

Filed hereditary amyloidosis 1 milestone
Filed EU (EMA) — hereditary amyloidosis 2021-09-13

“today announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vutrisiran” alnylam.com ↗

Approved Indication not stated 3 milestones
Label expansion US (FDA) AMVUTTRA — 2025-03-20 fda.gov ↗
Approved EU (EMA) Amvuttra — 2022-09-15 europa.eu ↗
Approved US (FDA) AMVUTTRA — 2022-06-13 fda.gov ↗

Trials 3

2019202020212022202320242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 33 trials · 3 met primary
Phase 3 NCT06679946 Dec 2024 → Mar 2028 expected — Alnylam Pharmaceuticals Enrolling by invitation Met primary
Phase 3 NCT04153149 Nov 2019 → May 2024 overdue — Alnylam Pharmaceuticals Active not recruiting Met primary
Phase 3 NCT03759379 Feb 2019 → Nov 2020 hereditary amyloidosis Alnylam Pharmaceuticals Completed Met primary
Comparator or background therapy2 trials
Phase 1 NCT02797847 Comparator Jun 2016 → Jan 2018 — Alnylam Pharmaceuticals Completed No outcome recorded
Phase 3 NCT07223203 Comparator Dec 2025 → Dec 2027 expected familial amyloid neuropathy Alnylam Pharmaceuticals Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 44

DateIssuerRelease
2026-08-30 Alnylam Pharmaceuticals Results Alnylam Presents New Data at ESC Congress 2026 Reinforcing Strength in RNAi-Powered TTR Silencing Across ATTR-CM Patient Populations and Treatment Settings alnylam.com ↗
The findings further reinforce the clinical profile of AMVUTTRA ® (vutrisiran) across transthyretin amyloidosis (ATTR) patient populations, treatment settings, and manifestations of disease.
2026-07-30 Alnylam Pharmaceuticals Results Alnylam Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Period Progress alnylam.com ↗
Presented New Data from HELIOS-B at Heart Failure 2026 Demonstrating Vutrisiran's Consistent Clinical Benefit Across Patient Populations
2026-05-12 Alnylam Pharmaceuticals Results New Data Analyses Presented at Heart Failure 2026 Demonstrate Robust and Consistent Clinical Benefit of Vutrisiran as a First-Line Treatment Option Across ATTR-CM Patient Populations, Including Patients with a High Disease Burden alnylam.com ↗
2026-04-30 Alnylam Pharmaceuticals Results Alnylam Pharmaceuticals Reports First Quarter 2026 Financial Results and Highlights Recent Period Progress alnylam.com ↗
New Data Presented at ACC.26 Further Support Use of Vutrisiran as First-Line Treatment for ATTR-CM
2026-03-30 Alnylam Pharmaceuticals Results New Data Presented at ACC.26 Further Support Benefits of Vutrisiran in ATTR-CM and Potential of Zilebesiran for Hypertension Management alnylam.com ↗
2025-11-08 Alnylam Pharmaceuticals Results New Post Hoc Analysis from the HELIOS-B Phase 3 Study Shows Vutrisiran Improved Measures of Heart Structure and Function in Patients with ATTR-CM alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results from new post hoc analyses of the HELIOS-B Phase 3 study of AMVUTTRA ® (vutrisiran), an RNAi therapeutic approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
2025-09-28 Alnylam Pharmaceuticals Results New Data from HELIOS-B Phase 3 Study Demonstrate Lower Rates of Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran alnylam.com ↗
Data from a post hoc analysis of the HELIOS-B study, which assessed whether treatment with vutrisiran was associated with a reduction in gastrointestinal (GI) adverse events in patients with ATTR-CM, compared to placebo, were presented during a late-breaking session at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2025 in Minneapolis, Minnesota .
2025-08-31 Alnylam Pharmaceuticals Results New Data From Landmark HELIOS-B Phase 3 Study Presented at ESC Congress 2025 Demonstrate Vutrisiran’s Long-Term Cardiovascular Benefit in ATTR-CM alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results of new analyses from the HELIOS-B Phase 3 study of AMVUTTRA ® (vutrisiran), an RNAi therapeutic approved for the treatment
2025-06-09 Alnylam Pharmaceuticals Regulatory Alnylam Receives European Commission Approval for AMVUTTRA® (vutrisiran) for the Treatment of ATTR Amyloidosis with Cardiomyopathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the European Commission (EC) has granted approval for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication for its orphan RNAi therapeutic AMVUTTRA ® (vutrisiran).
2025-05-17 Alnylam Pharmaceuticals Results AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B alnylam.com ↗
The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations.
2025-04-28 Alnylam Pharmaceuticals Regulatory Alnylam Receives Positive CHMP Opinion for Vutrisiran for the Treatment of ATTR Amyloidosis with Cardiomyopathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of its RNAi therapeutic vutrisiran for the treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
2025-03-29 Alnylam Pharmaceuticals Results Alnylam Presents New Data from the HELIOS-B Phase 3 Study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) at the American College of Cardiology’s Annual Scientific Session 2025 alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced moderated poster presentations of new data from the landmark HELIOS-B Phase 3 clinical trial, which evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a population representative of today’s patients.

All press releases naming this drug 56 releases

DateIssuerRelease

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 8 sources stand behind the page.

FieldValueCited text
Known as Vutrisiran ClinicalTrials.gov intervention name — accepted as the source's own label NCT07223203 ↗
3

NCT04153149 ↗

NCT06679946 ↗

NCT03759379 ↗

Known as ALN-65492 ChEMBL registry synonym — accepted as the source's own label CHEMBL4594511 ↗
Known as ALN-TTRSC02 ClinicalTrials.gov intervention name — accepted as the source's own label NCT02797847 ↗
Known as ALN-TTRSC02 FREE ACID ChEMBL registry synonym — accepted as the source's own label CHEMBL4594511 ↗
Known as Votrisiran ChEMBL registry synonym — accepted as the source's own label CHEMBL4594511 ↗
Action Inhibit “reduces hepatic production of transthyretin” PMID 37523143 ↗ Jul 2023
Modality Small interfering RNA (siRNA) “Vutrisiran, a subcutaneously administered RNA interference (RNAi) therapeutic” PMID 37523143 ↗ Jul 2023
Route Subcutaneous “Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection” NCT02797847 ↗
Target TTR “TTR reduction with vutrisiran Q3M” PMID 35875890 ↗ Jul 2022