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).
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 email@example.com.