Expanded Access Program
Expanded Access Program to Help Eligible Patients with hATTR Amyloidosis with Polyneuropathy Gain Access to Patisiran
The Expanded Access Program (EAP) provides treatment with patisiran*, an investigational medicine, to eligible adults who have a genotype-confirmed diagnosis of symptomatic hereditary transthyretin (hATTR) amyloidosis with polyneuropathy.
Physicians seeking access to patisiran for a patient via the EAP should submit their request to EAP@alnylam.com. Additional information can be found by going to www.ClinicalTrials.gov and searching NCT02939820.
*Patisiran is an investigational medicine that has not been approved by the Food and Drug Administration (FDA) or other health authorities.
About EAP for Patisiran
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:
- Having been diagnosed with hATTR amyloidosis with polyneuropathy, with a documented TTR mutation
- Having a New York Heart Association heart failure classification < 2
- Must not have participated in an interventional hATTR clinical trial within the last 24 months
- Are not currently eligible to participate in, or are currently enrolled in, an ongoing interventional hATTR clinical trial
- Do not have a known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
- Have no known serious comorbidities and must deemed to be fit for the program by your physician
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.
Frequently Asked Questions
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 early 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.