Compassionate Use / Expanded Access

Compassionate Use / Expanded Access

Expanded access, also called “compassionate use,” is the use of an investigational medicine prior to regulatory approval and outside of a clinical trial.

Akcea’s Expanded and Early Access Policy

Akcea Therapeutics’ mission is to develop and commercialize safe and effective RNA-targeting drugs for serious and rare diseases to address significant unmet needs in lipid-driven cardiometabolic disease and ATTR amyloidosis.  We do this by conducting clinical trials in which we collect safety and efficacy data about our experimental drugs with the goal of submitting those data to regulatory authorities, like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), so that these experimental drugs can be approved for use by patients.  For more information about clinical trials, including those being conducted by Akcea Therapeutics, please visit

At this time, participation in one of our clinical trials is generally the most appropriate way to access our experimental drugs.  However, in very rare circumstances, Akcea Therapeutics is able to make an experimental drug that we are developing available for patients outside of a traditional clinical trial under an expanded or early access protocol, sometimes referred to as a treatment protocol.  This is a program where patients may receive the experimental drug for as long as they meet certain criteria and are informed about the risks of participation.

In deciding whether to establish an expanded access protocol, Akcea will evaluate whether:

Current Expanded Access Protocols:

Early Access Program for volanesorsen in Patients with Familial Chylomicronemia Syndrome (FCS).

For more information about this expanded access protocol, please visit email, or call 1-833-EAP-VOLA (1-833-327-8652). Note: This Policy will be updated with the link to the listing for the volanesorsen Early Access Program once the listing is posted.

The existence of an expanded access protocol does not guarantee any individual patient access to any specific experimental drug and, at this time, participation in one of our clinical trials is generally the most appropriate way to access our investigational drugs.  If you have questions about an Akcea-sponsored clinical trial or our expanded access protocol, please speak with your physician or contact a study site listed on

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


Akcea Therapeutics has been acquired by Ionis Pharmaceuticals. For continued news and information about Akcea Therapeutics, please visit You are being redirected, please wait. If you have not been redirected in 30 seconds, please click the button below.

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