Expanded access programs (EAP) are a means by which investigational treatments 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.
Not every patient who wishes to take part in the EAP for patisiran will meet the entry criteria.
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.
Patisiran is an investigational medicine that uses the body’s natural processes to lower the levels of the TTR protein that causes TTR amyloidosis. It does so by targeting and silencing specific messenger RNA, blocking the production of TTR protein before it is made. This may help to reduce the level of TTR protein in the body. The safety and efficacy of patisiran are currently being studied in a phase 3 clinical study which is evaluating whether reducing TTR protein in the body may slow or halt the progression of hATTR amyloidosis with polyneuropathy.
To be eligible for the EAP, participants must meet certain requirements, including, but not limited to:
If you are a patient who is interested in enrolling in the EAP, please speak with your physician.
If you are a physician who is seeking to potentially enroll a patient in the EAP, please submit a request to EAP@alnylam.com.
1. What is the purpose of an Expanded Access Program (EAP)?
Expanded 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 this extended access program?
Patients 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, 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.