Helping Eligible Patients Gain Access to Alnylam Investigational Therapies

Alnylam is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families, and patient communities through education, empathy, and awareness. We understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Alnylam will consider providing a requesting physician with pre-approval access to a specific Alnylam investigational medicine, for the treatment of an individual patient outside of a clinical trial, or through an Alnylam early access program when certain conditions are met.


Learn More About Alnylam’s Compassionate Use Policy


Early Access Programs

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.


Patients meeting the eligibility requirements for an early access program (EAP) may be candidates to receive therapy; however, Alnylam cannot guarantee access to its investigational medicines. In addition to meeting eligibility requirements, a patient must be under the care of a physician trained on the administration of the drug at a selected medical establishment, and must provide informed consent to participate in the program. While Alnylam intends to ensure there is enough drug available to continue its early access programs for qualifying patients for each program’s duration, according to the relevant 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 any early access program at any time.


An early access program ends once the investigational therapy has been approved and is commercially available in the patient’s country.


For more information about early access, please contact Alnylam at


Givosiran for the treatment of acute hepatic porphyria (AHP)  Learn More ›


Patisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis  Learn More ›


Lumasiran for the treatment of primary hyperoxaluria type 1 (PH1)  Learn More ›


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