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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.
Read the full article on WikipediaHereditary transthyretin amyloidosis Clinical criteria: Treatment Phase: Initial treatment Patient must not have previously received PBS-subsidised treatment with this drug for this PBS indication, AND The condition must be hereditary transthyretin amyloidosis confirmed by genetic testing, AND Patient must have a Polyneuropathy Disability (PND) score description of either I, II, IIIA, IIIB; OR Patient must have a Familial Amyloid Polyneuropathy (FAP) stage description of 1 or 2, AND Patient must not have previously undergone a liver transplant, AND Patient must not exhibit heart failure symptoms (defined as New York Heart Association NYHA class III or IV). Treatment criteria: Must be treated by a consultant with experience in the management of amyloid disorders or in consultation with a consultant with experience in the management of amyloid disorders, AND Patient must be undergoing treatment with this drug as a monotherapy (i.e. not in combination with any other disease modifying medicines for amyloidosis disorders). Population criteria: Patient must have either: (i) stage 1 polyneuropathy, (ii) stage 2 polyneuropathy, AND Patient must be at least 18 years of age. PND scores in the context of this PBS restriction are: Stage 0 - No symptoms; Stage I - Sensory disturbances but preserved walking capability; Stage II - Impaired walking capacity but able to walk without stick or crutches; Stage IIIA - Walking with help of one stick or crutch; Stage IIIB - Walking with help of two sticks or crutches; Stage IV - Confined to wheelchair or bedridden. FAP stage in the context of this PBS restriction are: Stage 0 - No symptoms; Stage 1 - Unimpaired ambulation; Stage 2 - Assistance with ambulation required; Stage 3 - Wheelchair-bound or bedridden. Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail. If the application is submitted through HPOS form upload or mail, it must include: (a) details of the proposed prescription; and (b) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).
“Vutrisiran is a gene silencing double-stranded siRNA-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through RNA interference by binding and silencing messenger RNA (mRNA) encoding for disease causing protein (Transthyretin), which results in a reduction of serum TTR protein and TTR protein deposits in tissues. Vutrisiran utilises a GalNAc conjugate delivery platform which is an enhanced stabilisation chemistry. This allows subcutaneous administration of smaller doses with longer dosing intervals.”
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