Medical Professionals
US EARLY ACCESS PROGRAM FOR PATISIRAN
The patisiran US Expanded Access Program (EAP) is an Alnylam program which aims to provide access to patisiran for patients with ATTR amyloidosis with cardiomyopathy who have had an inadequate response to or cannot tolerate currently available treatment.
Eligibility Requirements
To be eligible to seek access to patisiran through the early access program in the United States, participants must meet certain requirements, including, but not limited to:
Inclusion Criteria
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Age 18 (or age of legal consent, whichever is older) to 85 years, inclusive.
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Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy; AND
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Had an inadequate response to or could not tolerate standard of care, in the opinion of the investigator.
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- Is not eligible for on-label use of commercial patisiran in the opinion of the investigator.
Exclusion criteria:
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NYHA Class IV
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NYHA Class III AND ATTR amyloidosis disease Stage 3 (defined as both NT-proBNP >3000 ng/L and estimated glomerular filtration rate [eGFR] <45 ml/min/1.73 m2). [Gillmore 2018]
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Current or future participation in another investigational device or drug study, scheduled to occur during this study, or has received an investigational agent or device within 30 days (or 5 half-lives of the investigational drug, whichever is longer) prior to dosing (Day 1). Patients who have previously participated in a gene therapy trial for hATTR amyloidosis.
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Patients currently enrolled in, eligible for inclusion in, or who have dropped out of an ongoing interventional (therapeutic) clinical trial related to ATTR amyloidosis.
If you are a patient living in the United States and are interested in seeking access to patisiran through the US 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 US physician who is seeking to obtain access to patisiran for a patient in your care, please contact Alnylam at EAP@alnylam.com.
If you are a physician or patient and have a medical question, please contact medinfo@alnylam.com.
