ATTR Expanded Access Program (EAP) by Ionis

Physicians who would like to enroll a patient in the ATTR EAP by Ionis should submit their request to or call 1-833-ATTR-EAP (1-833-288-7237). For more information about this expanded access program, please visit (NCT03400098).

About the EAP

When a patient with a serious illness has exhausted all treatment options and is unable to enter a clinical trial, the US Food and Drug Administration (FDA) may allow the patient to be treated with an investigational drug that is not yet approved for use. Sometimes referred to as “compassionate use,” EAPs help provide these patients with the treatment that they would otherwise not be able to receive.

It’s important to note that clinical trials are still the safest route for providing patients access to investigational treatments because they are well controlled and usually include a large number of patients.

To learn more about available clinical trials, visit

What are the eligibility requirements?

Unfortunately, not every patient who would like to take part is eligible for the ATTR EAP by Ionis. According to the FDA, participants must meet the following requirements to enroll:

  • The patient and a licensed physician are both willing to participate
  • The patient’s physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition
  • The probable risk to the patient from the investigational product is not greater than the probable risk from the disease or condition
  • The patient is unable to obtain the investigational drug under an investigational new drug (IND) application and is, therefore, unable to participate in a clinical trial

For more information, please visit the FDA’s Expanded Access (Compassionate Use) page.

How do I enroll?

If you are a patient looking to enroll in the ATTR EAP by Ionis, or if you have any questions, please speak with your physician.

If you are a physician looking to enroll a patient in the ATTR EAP by Ionis, please submit a request to or call 1-833-ATTR-EAP (1-833-288-7327).

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