Although patisiran is approved in the U.S., it is still an investigational treatment in countries outside of the U.S. Early Access is still available in other countries. Physicians seeking access to patisiran for a patient outside of the U.S. via the EAP should submit their request to EAP@alnylam.com.
Not every patient who wishes to take part in the EAP for patisiran will meet the entry criteria.
To be eligible for the EAP, participants must meet certain requirements, including, but not limited to:
As with any investigational medicine that has not been approved by regulatory authorities, patisiran may or may not be effective in treating hATTR amyloidosis with polyneuropathy and there may be risks associated with its use. 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 outside of the U.S. who is interested in enrolling in the EAP, please speak with your physician.
If you are a physician outside of the U.S. who is seeking to potentially enroll a patient in the EAP, please submit a request to EAP@alnylam.com.
Patisiran is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is currently available in the United States and marketed as ONPATTRO. In certain other countries, patisiran is an investigational treatment. The safety and efficacy of patisiran have not been evaluated by the EMA or any other health authorities outside the U.S.
1. What is the purpose of an Early Access Program (EAP)?
Early access programs (EAP) are a means by which investigational drugs are made available, in certain circumstances, to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited.
2. How does the EAP request process work?
Physicians seeking access to patisiran via the EAP for patients with no alternative treatment options should submit their requests to EAP@alnylam.com. Alnylam regularly monitors this mailbox and will typically respond within three business days of receipt.
Individual patients interested in learning more should work with their physician to discuss potential benefits and risks and determine if they might be eligible. Requests must be submitted by a physician.
3. Is everyone diagnosed with hATTR amyloidosis with polyneuropathy eligible for the early access program?
Patients outside the U.S. meeting the eligibility requirements for the EAP may be candidates to receive patisiran, but Alnylam cannot guarantee access to patisiran through the program. In addition to meeting the inclusion/exclusion criteria, a patient must agree to participate by understanding and signing the Informed Consent Form, and must be under the care of a physician trained on the administration of the drug at a selected institution. While Alnylam intends to ensure there is sufficient drug supply to continue the program for qualifying patients for its duration, and according to each country’s local regulatory requirements, Alnylam cannot guarantee drug supply. For this and potentially other reasons that are currently unforeseeable, Alnylam reserves the right to restrict access or discontinue the program at any time.