Criteria
Coverage is subject to the specific terms of the member's benefit plan.
The use of patisiran (Onpattro) may be considered medically necessary for the treatment of individuals 18 years of age and older with a diagnosis of hereditary TTR amyloidosis when ALL of the following criteria are met:
- The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
- The requested medication must be prescribed by, or in consult with, a neurologist, geneticist, or specialist in the treatment of amyloidosis; and
- The diagnosis must be confirmed by both of the following:
- Genetic testing confirming a pathogenic TTR mutation (e.g., V30M); and
- Amyloid deposits via tissue biopsy; and
- Documentation of ONE of the following must be provided:
- Baseline polyneuropathy disability (PND) score less than or equal to IIIb; or
- Baseline Coutinho staging system stage 1 or 2; or
- Baseline Neuropathy Impairment Score [NIS] of 5-130; or
- Karnofsky Performance Status score of ≥60%; and
- The individual has not had a liver transplant; and
- The individual has clinical signs and symptoms of the disease (e.g., peripheral neuropathy, numbness, altered pain and temperature sensation, decreased pinprick sensation); and
- The individual is not receiving any other TTR reducing agent (i.e., vutrisiran, patisiran, tafamidis, inotersen, eplontersen).
Reauthorization Criteria
Continuation of therapy with patisiran (Onpattro) may be considered medically necessary when the following is met:
- Documentation of a therapeutic response as evidenced by stabilization or improvement (e.g., improved neurologic impairment, motor function, quality of life, slowing of disease progression, etc.) from baseline in ONE of the following:
- Polyneuropathy disability (PND) score less than or equal to IIIb; or
- Coutinho staging system stage 1 or 2; or
- Baseline Neuropathy Impairment Score [NIS] of 5-130; or
- Karnofsky Performance Status score of ≥60%.
The use of patisiran (Onpattro) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.
Procedure Codes
The use of Vutrisiran (Amvuttra) may be considered medically necessary for the treatment of individuals 18 years of age and older when ALL of the following criteria are met:
Polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN)
The use of Vutrisiran (Amvuttra) may be considered medically necessary for the treatment of individuals 18 years of age and older with a diagnosis of hereditary TTR amyloidosis when ALL of the following criteria are met:
- The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
- The requested medication must be prescribed by, or in consult with, a neurologist, geneticist, or specialist in the treatment of amyloidosis; and
- The diagnosis must be confirmed by both of the following:
- Genetic testing confirming a pathogenic TTR mutation (e.g., V30M); and
- Amyloid deposits via tissue biopsy; and
- Documentation of ONE of the following must be provided:
- Baseline polyneuropathy disability (PND) score less than or equal to IIIb; or
- Baseline Coutinho staging system stage 1 or 2; or
- Baseline Neuropathy Impairment Score [NIS] of 5-130; or
- Karnofsky Performance Status score of ≥60%; and
- The individual has not had a liver transplant; and
- The individual has clinical signs and symptoms of the disease (e.g., peripheral neuropathy, numbness, altered pain and temperature sensation, decreased pinprick sensation); and
- The individual is not receiving any other TTR reducing agent (i.e., vutrisiran, patisiran, tafamidis, inotersen, eplontersen).
Reauthorization Criteria
Continuation of therapy with Vutrisiran (Amvuttra) for diagnosis of hereditary TTR amyloidosis may be considered medically necessary when the following is met:
- Documentation of a therapeutic response as evidenced by stabilization or improvement (e.g., improved neurologic impairment, motor function, quality of life, slowing of disease progression, etc.) from baseline in ONE of the following:
- Polyneuropathy disability (PND) score less than or equal to IIIb; or
- Coutinho staging system stage 1 or 2; or
- Baseline Neuropathy Impairment Score [NIS] of 5-130; or
- Karnofsky Performance Status score of ≥60%.
Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM)
The use of Vutrisiran (Amvuttra) may be considered medically necessary for the treatment of individuals 18 years of age and older with a diagnosis of cardiomyopathy transthyretin-mediated amyloidosis (ATTR-CM) when ALL of the following criteria are met:
- The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
- The requested medication must be prescribed by, or in consult with, a cardiologist, geneticist, or specialist in the treatment of amyloidosis; and
- Confirmation of the diagnosis by both of the following must be provided:
- presence of grade 2 or 3 positive bone tracer cardiac scintigraphy; and
- absence of monoclonal protein confirmed by serum protein immunofixation, urine protein immunofixation, or serum free light chain ratio analysis; and
- The individual must have heart failure class I or II with at least 1 prior hospitalization for heart failure or with symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) despite 6-months of adherent use of a diuretic; and
- The individual has an end-diastolic interventricular septal wall thickness of at least 12 mm; and
- The individual must not have any of the following:
- eGFR less than 25 mL/min/1.73m; and
- NYHA class IV symptoms or severe aortic stenosis; and
- Previous heart transplant or implanted cardiac mechanical assist device; and
- Previous liver transplant; and
- Baseline 6MWT > 100 meters must be submitted; and
- The individual is not receiving any other TTR reducing agent (i.e., patisiran, elplontersen); and
- The individual must have failed a 90-day trial of each Attruby and Vyndaqel/Vyndamax, as evidenced by paid claims or pharmacy printouts.
Reauthorization Criteria
Continuation of therapy with Vutrisiran (Amvuttra) for diagnosis of cardiomyopathy transthyretin-mediated amyloidosis (ATTR-CM) may be considered medically necessary when the following is met:
- The individual has received a therapeutic response as evidenced by stabilization or improvement from baseline in both of the following:
The use of Vutrisiran (Amvuttra) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.
Procedure Codes
