Initial Treatment – Hereditary Transthyretin-Mediated Amyloidosis Polyneuropathy
Patisiran, inotersen, and vutrisiran are considered medically necessary for individuals if they meet criteria 1 through 5:
18 years of age or older.
Confirmatory diagnosis of hATTR by a genetic test OR tissue biopsy showing amyloid deposition.
Presence of clinical signs and symptoms of polyneuropathy characterized by any one of the following:
Baseline polyneuropathy disability (PND) IIIb or lower (see Table 1 in background section)
Baseline familial amyloid polyneuropathy (FAP) Stage 1 or 2 (see Table 1 in background section).
Does not have ANY of the following:
New York Heart Association (NYHA) class III or IV heart failure
Sensorimotor or autonomic neuropathy not related to hATTR amyloidosis (monoclonal gammopathy, autoimmune disease, etc.)
Prior liver transplantation.
Does not have any U.S. Food and Drug Administration (FDA) labeled contraindications to the requested agent and is intended to be used consistently with the FDA approved label (see policy guidelines).
Initial authorization period is for 12 months.
Continuation of Treatment – Hereditary Transthyretin-Mediated Amyloidosis Polyneuropathy
Incremental reauthorization of patisiran, inotersen, and vutrisiranare considered medically necessary for individuals if they meet criteria 1 through 2:
Continues to meet the initial treatment criteria cited above.
Documentation of stabilization OR improvement via use of objective measurements, such as 10-MWT, COMPASS-31, PND Score or 5 EQ-5D.
Reauthorization period is for 12 months.
Patisiran, inotersen, and vutrisiran are considered investigational when the above criteria are not met.
Initial Treatment – Hereditary Transthyretin-Mediated Amyloidosis Cardiomyopathy
Tafamidis and tafamidis meglumine are considered medically necessary for individuals if they meet criteria 1 through 5:
18 years of age or older.
Confirmatory diagnosis of hereditary or wild-type transthyretin-mediated amyloidosis by a genetic test OR tissue biopsy showing amyloid deposition.
Presence of clinical signs and symptoms of cardiac involvement by all of the following:
End-diastolic interventricular septal wall thickness exceeding 12 mm on echocardiography.
History of heart failure, with at least one prior hospitalization for heart failure or clinical evidence of heart failure (without hospitalization) manifested in signs or symptoms of volume overload or elevated intracardiac pressures requiring treatment with a diuretic for improvement.
Baseline NT-proBNP ≥ 600 pg/mL.
Does not have any of the following:
NYHA class IV heart failure.
Presence of light-chain amyloidosis.
History of liver or heart transplantation.
Implanted cardiac device.
Does not have any U.S. FDA labeled contraindications to the requested agent and is intended to be used consistently with the FDA approved label (see policy guidelines).
Initial authorization period is for 12 months.
Continuation of Treatment – Hereditary Transthyretin-mediated Amyloidosis Cardiomyopathy
Incremental reauthorization for tafamidis and tafamidis meglumine are considered medically necessary for individuals if they meet criteria 1 through 2:
Continues to meet the initial treatment criteria cited above.
Documentation of stabilization OR improvement via use of objective measurements, such as number of hospitalizations, 6-MWT, or KCCQ-OS.
Reauthorization period is for 12 months.
Tafamidis and tafamidis meglumine are considered investigational when the above criteria are not met.
