Amvuttra

Vutrisiran

• Brand(s): Amvuttra
• Dosage Form/Strength: 25 mg/0.5 mL prefilled syringe
• Effective date: December 13, 2024

Initiation criteria:

For the treatment of patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN), meeting all the following criteria:

1. Age 18 years of age or older;
   AND
2. Has a confirmed genetic diagnosis of hereditary transthyretin-mediated amyloidosis;
   AND
3. Symptomatic with Polyneuropathy disability stage I to ≤ IIIB or with Familial amyloidotic polyneuropathy stage I or II.
4. Under the care of a specialist with experience in the diagnosis and management of hATTR-PN

Exclusion Criteria:

• Pre-symptomatic patients.
• Patients diagnosed with severe heart failure symptoms (defined as New York Heart Association class III or IV).
• Patients who are recipients of a liver transplant.
• Patients who will be using vutrisiran in combination with other interfering ribonucleic acid drugs or transthyretin stabilizers used to treat hATTR.

Discontinuation criteria: Treatment with vutrisiran will be discontinued for patients who are:

• Permanently bedridden and dependent on assistance for basic activities of daily living, or
• Receiving end-of-life/palliative care where survival of less than one year is expected.

Renewal Criteria:

Renewal of funding will be considered if patients do not meet the discontinuation criteria.

Dosage:

25 mg injected subcutaneously once every 3 months.

Patients should be assessed after 9 months of treatment and then every six months thereafter.

Duration of Approval of initiation requests: 10 months

Duration of Approval of first renewal: 6 months

Duration of Approval of 2nd and subsequent renewals: 1 year

Notes to Prescribers:

• Laboratory documentation for the genetic mutation for hATTR must be included with the application.
• Signs and symptoms of polyneuropathy should be listed.
• In your application please list all drugs that the patient is using including whether he/she is using any of the following: Diflunisal, Inotersen, Patisiran, Tafamidis
• Confirmation that the patient does not meet each of the listed exclusions must be provided on the request.

Definitions:

Familial Amyloid Polyneuropathy (FAP) stage: Clinical staging system for the neuropathy symptoms of hATTR (formerly termed familial amyloid neuropathy).

• FAP Stage 1: Walking without assistance, mild neuropathy (sensory, autonomic, and motor) in lower limbs
• FAP Stage 2: Walking with assistance, moderate impairment in lower limbs, trunk, and upper limbs
• FAP Stage 3: wheelchair or bed-ridden, severe neuropathy

Polyneuropathy disability score (PND): A five-stage measure of neuropathy impairment ranging from 0 (no impairment) to 4 (confined to a wheelchair or bedridden).

• Stage 0: no impairment
• Stage I: sensory disturbances but preserved walking capability
• Stage II: impaired walking capability but ability to walk without a stick or crutches
• Stage IIIA: walking only with the help of one stick or crutch
• Stage IIIB: walking with the help of two sticks or crutches
• Stage IV: confined to a wheelchair or bedridden

EAP Drug Request Form:

Standard Form for EAP Drug Requests