Overview
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mutations have been identified so far, but the most common one is the replacement of valine with methionine at position 30 (Val30Met). The Val30Met variant is the most prevalent among hereditary ATTR patients with polyneuropathy, especially in Portugal, France, Sweden, and Japan. TTR mutations lead to the formation of misfolded TTR proteins, which form amyloid fibrils that deposit in different types of tissues. By targeting TTR mRNA, vutrisiran reduces the serum levels of TTR. Vutrisiran is commercially available as a conjugate of N-acetylgalactosamine (GalNAc), a residue that enables the delivery of siRNA to hepatocytes. This delivery platform gives vutrisiran high potency and metabolic stability, and allows for subcutaneous injections to take place once every three months. Another siRNA indicated for the treatment of polyneuropathy associated with hereditary ATTR is patisiran. Vutrisiran was approved by the FDA in June 2022.
Indication
Vutrisiran is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Associated Conditions
- Polyneuropathies
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
---|---|---|---|---|---|
2024/11/08 | Phase 3 | ENROLLING_BY_INVITATION | |||
2019/11/06 | Phase 3 | Active, not recruiting | |||
2018/11/30 | Phase 3 | Active, not recruiting | |||
2016/06/14 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
---|---|---|---|---|---|
Alnylam Pharmaceuticals, Inc. | 71336-1003 | SUBCUTANEOUS | 25 mg in 0.5 mL | 8/19/2022 |
EMA Drug Approvals
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No EMA approvals found for this drug. |
HSA Drug Approvals
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No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
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No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
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No PPB approvals found for this drug. |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
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No TGA approvals found for this drug. |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
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No Health Canada approvals found for this drug. |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
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AMVUTTRA 25 MG SOLUCION INYECTABLE EN JERINGA PRECARGADA | 1221681001 | SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Philippines FDA approvals found for this drug. |
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Saudi SFDA approvals found for this drug. |
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
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No Malaysia NPRA approvals found for this drug. |
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
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No UK EMC drug information found for this drug. |
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