Expanded Access Programs

We are committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. Arrowhead’s Expanded Access program provides a pathway for certain patients with serious diseases, who are ineligible or unable to participate in an ongoing Arrowhead clinical trial, to receive access to an investigational medicine outside a clinical study. As with any therapy that has not been approved by regulatory authorities, treatment provided through Expanded Access may or may not be effective in treating your condition, and there may be risks associated with its use. If you are a patient interested in participating, you should talk to your physician about the potential benefits and risks of receiving an unapproved treatment through this program. To learn more about access to investigational medicines at Arrowhead, review our compassionate use agreement.

Plozasiran Expanded Access Program (EAP) for Familial Chylomicronemia Syndrome (FCS) and High-Risk Severe Hypertriglyceridemia (SHTG) Now Available

Plozasiran is a medicine designed to reduce the production of the protein Apolipoprotein-CIII (ApoC3) through the natural RNA interference (RNAi) mechanism.  ApoC3 is a protein that is produced in liver cells and inhibits the formation and clearance of various lipids and lipoproteins, including triglycerides. Plozasiran remains investigational for SHTG and pediatric FCS populations in which it has not yet been proven as safe and effective to reduce the level of ApoC3, thereby reducing triglycerides. Plozasiran is approved in the U.S. as an adjunct to diet to reduce triglycerides in adults with FCS.  The most common adverse reactions as included in the US prescribing information are hyperglycemia, headache, nausea, and injection site reactions. The plozasiran FCS EAP is for individuals under 18 years old living with familial chylomicronemia syndrome (FCS). The plozasiran SHTG EAP is for individuals with severe hypertriglyceridemia at high risk of acute pancreatitis events.

If you are a patient or caregiver wishing to know more about the plozasiran EAP for FCS or high-risk SHTG, please discuss the EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the plozasiran EAP or would like to request access for a patient, please contact  EAP@arrowheadpharma.com.