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 http://akceatx.com/health-care-professionals/clinical-trials/.
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:
- there is substantial scientific evidence to support both the safety and the efficacy of an experimental drug for the proposed use, typically after positive Phase 3 data is available;
- it has been established that expanded access will not interfere with the conduct of clinical trials or other aspects of development that could impact marketing approval;
- there is adequate supply of the experimental drug; and
- the experimental drug can be administered, and it is logistically feasible to make it available, outside of the clinical trial setting.
Current Expanded Access Protocols:
Expanded Access Program for inotersen in Patients with Hereditary Transthyretin Amyloidosis (hATTR).
Early Access Program for volanesorsen in Patients with Familial Chylomicronemia Syndrome (FCS).
For more information about this early access program, please email VolanesorsenEAP@akceatx.com, or call 1-833-EAP-VOLA (1-833-327-8652). Note: This Policy will be updated with the link to the clinicaltrial.gov 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 http://akceatx.com/our-programs/pipeline/.
For more information, please visit the FDA’s Expanded Access (Compassionate Use) page: https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm