Expanded Access Policy

Expanded Access to Investigational Therapies Policy

Voyager’s mission is to develop and deliver life-changing therapies to people around the world living with severe neurological diseases. This begins with our commitment to discovery, research, development and manufacturing, and extends to our engagement with patients, healthcare providers, caregivers and advocacy organizations.

Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational treatments and medical devices for serious diseases or conditions. Expanded access refers to the use of an investigational therapy (one that has not been approved by the U.S. Food & Drug Administration (FDA) or other applicable regulatory authority) outside of a clinical trial where more comprehensive safety and efficacy data are collected.

When considering expanded access, it is important to consider guidelines from the U.S. FDA as well as guidelines from regulatory agencies outside of the U.S. According to the FDA, expanded access may be appropriate when all of the following apply:

  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition; 
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
  • Patient enrollment in a clinical trial is not possible; 
  • Potential patient benefit based on available safety and efficacy information justifies the potential risks of treatment;
  • Providing the investigational medical product will not interfere with, or delay, investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

Currently, Voyager does not offer an expanded access program. Our goal is to provide patients with access to the gene therapy programs that we are developing as safely and quickly as possible. We believe the most appropriate way to achieve this goal is through participation in our clinical trials. In accordance with the 21st Century Cures Act, Voyager may revise this policy at any time.

If you have questions about Voyager’s expanded access policy, please contact Voyager Therapeutics at expandedaccess@vygr.com. We plan to respond within 5 business days of receiving your question.