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Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide.
Read the full article on WikipediaHereditary transthyretin amyloidosis Clinical criteria: Treatment Phase: Initial treatment Population criteria: Patient must have either: (i) stage 1 polyneuropathy, (ii) stage 2 polyneuropathy. Patient must not have previously received PBS-subsidised treatment with this drug for this PBS indication. Population criteria: Patient must be at least 18 years of age. 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). 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).
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