Early Access Program to Help Eligible Patients with hATTR Amyloidosis with Polyneuropathy Gain Access to Patisiran

The Early Access Program (EAP) provides treatment with patisiran to eligible adults who have a genotype-confirmed diagnosis of hereditary transthyretin (hATTR) amyloidosis with symptomatic polyneuropathy. Early access programs 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.


Enrollment in EAP ends once the investigational therapy has been approved and is commercially available in the patient’s country. Patisiran was approved by the U.S. Food and Drug Administration (FDA) as ONPATTRO® (patisiran) lipid complex injection on August 10, 2018 for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults; therefore, the EAP is closed in the U.S. and is not enrolling additional patients. Current EAP participants in the U.S. who would like to remain on therapy should discuss next steps with their physician. Patients and healthcare professionals may visit www.AlnylamAssist.com or call 1-833-256-2748 to learn more about starting treatment with ONPATTRO.

About EAP for Patisiran

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.


Eligibility Requirements

To be eligible for the EAP, 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 established through neurologic consultation
  • Having a New York Heart Association Heart Failure Classification ≤ 2
  • Must not have participated in an interventional 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
  • Have no known serious comorbidities and must deemed to be fit for the program by treating physician


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.



For more information about the EAP, please contact Alnylam at +1.617.715.0200 or EAP@alnylam.com.


About Patisiran

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.


Frequently Asked Questions

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.


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