Aevi Genomic Medicine is focused on translating genetic discoveries into novel therapies to improve the lives of children and adults with pediatric onset life altering diseases. These novel therapies are tested in clinical trials to study the safety and effectiveness of the investigational treatment. The information from the clinical trials may be used to support an application to health authorities to market the medication. If that application is approved, the medicine may be prescribed to patients.
Expanded access (also referred to as compassionate use) provides a process for patients to gain access to an investigational treatment for a serious disease or condition. An investigational treatment has not yet been demonstrated to be safe and effective and has not been approved by the FDA.
At this time, Aevi Genomic Medicine encourages participation in one of our clinical trials where some participants may access our investigational therapies. To get more information about available Aevi Genomic Medicine clinical trials, visit Aevi Clinical Trials (at clinicaltrials.gov).
As part of our commitment to patients, Aevi Genomic Medicine may implement an expanded access program for an investigational treatment for a serious or life-threatening condition.
The decision to allow expanded access to an investigational treatment is complex and Aevi Genomic Medicine’s policy is aligned with guidelines from FDA and other regulatory authorities. Consideration of a request for expanded access to an investigational treatment may be granted only if each of the following criteria are met:
Aevi Genomic Medicine may revise this policy at any time and will remain aligned with the 21st Century Cures Act. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov after such record becomes active.