Hereditary transthyretin-mediated (hATTR) amyloidosis: a multisystem, often fatal disease1-3

Peripheral sensory-motor neuropathy
  • Neuropathic pain
  • Paresthesia
  • Weakness
  • Bilateral carpal tunnel syndrome
  • Difficulty walking
Autonomic neuropathy
  • Orthostatic hypotension
  • Urinary retention
  • Recurrent urinary tract infections
  • Sexual dysfunction
  • Sweating abnormalities
Gastrointestinal manifestations
  • Diarrhea
  • Nausea
  • Vomiting
  • Unintentional weight loss
Cardiovascular manifestations
  • Arrhythmias
  • Conduction abnormalities
  • Heart failure

ONPATTRO® (patisiran) does not treat all of the symptoms of hATTR amyloidosis.

hATTR amyloidosis can progress quickly and lead to significant burden4-6

Person with cane
Decreased ambulation5
  • Inability to walk unaided
  • Wheelchair-bound or bedridden
Person standing
Decline in daily function6,7
  • Impairment in self-care
  • Impairment in ability to perform usual activities
  • Pain/discomfort
Rain cloud
Social burden8
  • Anxiety
  • Depression
Premature death9
  • hATTR amyloidosis can lead to premature death in 4.7 yearsa

aMedian survival following diagnosis.

Important Safety Information and Indication

Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO® (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).


ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

For additional information about ONPATTRO, please see the full Prescribing Information.


1. Adams D, Coelho T, Obici L, et al. Neurology. 2015;85(8):675-682.

2. Conceição I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21(1):5-9.

3. Shin SC, Robinson-Papp J. Mt Sinai J Med. 2012;79(6):733-748.

4. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

5. Coutinho P, Martins da Silva A, Lopes Lima JL, et al. Excerpta Medica; 1980:88-98.

6. Vinik EJ, Vinik AI, Paulson JF, et al. J Peripher Nerv Syst. 2014;19:104-119.

7. Pruppers MHJ, Merkies ISJ, Faber CG, et al. J Peripher Nerv Syst. 2015;20(3):319-327.

8. Lopes A, Fonseca I, Sousa A, et al. Amyloid. 2018;25(1):26-36.

9. Swiecicki PL, Zhen DB, Mauermann ML, et al. Amyloid. 2015;22(2):123-131.