This website will enable you to register yourself and your institution and submit your Compassionate Use request to Alnylam for review as well as track the progress of your existing request.
Please reference Alnylam’s Compassionate Use Policy below:
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.
Consistent with Alnylam’s mission to bring innovative medicines to patients with serious or potentially life-threatening illnesses or conditions, we are focused on conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical physicians and with patients who participate in our studies to develop new, safe and effective therapies. At the same time, 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 access to a specific Alnylam investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following but are not limited to:
- The patient has a serious or potentially life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
- The investigational drug is in active clinical development or has at least one active marketing authorization with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
- A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
- Making the investigational drug available will not negatively impact or significantly delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
- Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound is different and Alnylam may decide not to make an investigational drug available under this policy. Furthermore, the fact that an investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to requests for other patients whose circumstances and medical histories may be different. Requests will be considered on a case-by-case basis.
Alnylam is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician and Alnylam may require more detailed medical information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Alnylam whose decisions are final.
Physicians seeking access to Alnylam investigational medicines for patients with no alternative treatment options should submit their requests via the Alnylam Compassionate Use portal at https://alnylam-compassionateuse.steeprockinc.com. Alnylam regularly monitors the Compassionate Use portal and will use our best efforts to acknowledge each submitted request within 3 business days after receipt.