vutrisiran
Sign in to saveAlso known as Amvuttra, ALN-65492
Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is a double stranded small interfering RNA (siRNA; also called RNA interference or RNAi therapeutic) that interferes with the expression of the transthyretin (TTR) gene. Transthyretin is a serum protein made in the liver whose major function is transport of vitamin A and thyroxine. Rare mutations in the transthyretin gene result in accumulation of large amyloid deposits of misfolded transthyretin molecules most promi
Clinical Trials
4 registered- ACTIVE_NOT_RECRUITINGMyocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or VutrisiranRennes University Hospital · NCT05873868
- PHASE3ACTIVE_NOT_RECRUITINGHELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyAlnylam Pharmaceuticals · NCT04153149
- PHASE3COMPLETEDHELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)Alnylam Pharmaceuticals · NCT03759379
- PHASE3ENROLLING_BY_INVITATIONA Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyAlnylam Pharmaceuticals · NCT06679946
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Encyclopedic overview
12 sectionsContents
- Medical uses
- Adverse effects
- Pharmacokinetics
- Mechanism of action
- History
- Efficacy and trials
- Safety
- Society and culture
- Legal status
- Names
- Formulations
- References
Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is a double stranded small interfering RNA (siRNA; also called RNA interference or RNAi therapeutic) that interferes with the expression of the transthyretin (TTR) gene. Transthyretin is a serum protein made in the liver whose major function is transport of vitamin A and thyroxine. Rare mutations in the transthyretin gene result in accumulation of large amyloid deposits of misfolded transthyretin molecules most prominently in peripheral nerves and the heart. Patients with hATTR typically present with polyneuropathy or autonomic dysfunction followed by cardiomyopathy which, if untreated, is fatal within 5 to 10 years.
Vutrisiran was approved for medical use in the United States in June 2022, in the European Union in September 2022, and in Australia in June 2024.
Excerpted from Wikipedia’s “vutrisiran” article, available under the CC BY-SA 4.0 licence.