Patisiran is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis. It is commercially available in the United States, Canada, and some European countries to treat the polyneuropathy of hATTR amyloidosis. Where it is approved, it is marketed as ONPATTRO.

Eligibility Requirements

To be eligible to seek access to patisiran through the early access program in a country where it is not commercially available, participants must meet certain requirements, including, but not limited to:

  • Having a documented TTR mutation and a confirmed diagnosis of symptomatic hATTR amyloidosis with polyneuropathy
  • Having a New York Heart Association Heart Failure Classification ≤ 2
  • Must not have participated in an hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
  • Are not currently eligible to participate in, or are currently enrolled in, an ongoing interventional hATTR amyloidosis clinical trial
  • Do not have known primary amyloidosis (amyloid light-chain [AL] amyloidosis) or leptomeningeal amyloidosis
  • Have no known serious comorbidities and are deemed fit for the program by a treating physician


If you are a patient interested in participating, you should talk to your physician about the potential benefits and risks of taking part in the program.


If you are a patient living in a country where patisiran is not commercially available and are interested in seeking access to patisiran through the early access program, please speak with your physician. Alnylam can only respond to a physician requesting access to patisiran on behalf of a patient in his/her care.


If you are a physician who is seeking to obtain access to patisiran for a patient in your care, please contact EAP@alnylam.com.


If you are a physician or patient and have a medical question, please contact medinfo@alnylam.com.


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