Expanded Access Policy

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).

How Expanded Access Works

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:

• The patient has a serious or life-threatening disease
• The patient is unable to participate in an ongoing clinical trial of that investigational treatment
• The patient's disease is similar to the indication(s) for which the investigational treatment is currently being studied
• No comparable therapies (investigational or approved) are available
• The potential benefits of the investigational treatment outweigh the potential risks and those potential risks are not unreasonable in the context of the disease or condition to be treated
• There is an adequate supply of the investigational treatment
• It is logistically feasible to make the investigational treatment available and it can be safely administered in a controlled setting
• The expanded access to the investigational treatment will not in any way compromise the planned development or interfere with the regulatory approval process

Aevi Genomic Medicine will evaluate each request for expanded access in a timely and fair manner. A request for expanded access to an investigational treatment must be made by a qualified and licensed physician to expandedaccess@aevigenomics.com. Aevi Genomic Medicine will acknowledge receipt of a request within 5 business days. Each request will be evaluated by key members of our medical and regulatory team, and a decision will be provided to the requesting healthcare provider within 2 weeks.

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.