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:
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 email@example.com. We plan to respond within 5 business days of receiving your question.
Voyager initiated RESTORE-1, a Phase 2, randomized, placebo-surgery controlled, double-blinded, multi-center clinical trial to evaluate the safety and efficacy of VY-AADC for the potential treatment of advancing Parkinson’s disease. Multiple clinical trial sites have been selected for participation in the RESTORE-1 trial.
RESTORE-1 is a clinical trial for people with advancing Parkinson’s disease. If your response to Parkinson’s disease medications has become unpredictable or isn’t what it used to be, this clinical trial may be an option that you should discuss with your doctor.
For more information about the RESTORE-1 clinical trial, including an eligibility questionnaire, please visit restore1study.com.