EARLY ACCESS PROGRAM FOR PATISIRAN

Patisiran is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) and is approved in >30 countries for the treatment of hATTR amyloidosis with polyneuropathy in adults. Specific indications vary by country/region. Where it is approved, it is marketed as ONPATTRO® (patisiran).

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 (AL amyloidosis) or leptomeningeal amyloidosis
• Having no known serious comorbidities and are deemed fit for the program by a treating physician

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 EAP, please speak with your physician. Alnylam can only respond to a physician requesting access to patisiran on behalf of a patient in their 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.