Penn Orphan Disease Center

The Orphan Disease Center (ODC) of the University of Pennsylvania (Penn) is led by Dr. Jim Wilson. The ODC was formed to facilitate and fund research and to develop transformative therapies for rare diseases with significant unmet need.

As part of our research collaboration with Penn's Gene Therapy Program, we have access to the ODC's insights and capabilities in the study of rare diseases. We leverage our close working relationship with the ODC to develop historical and prospective external data for each disease for use in building comparable patient profiles of participants in interventional trials. In addition, we believe the ODC's close ties to leading clinical centers for rare, monogenic central nervous system disorders will improve our ability to identify potential patients for trial enrollment, and enhance patient retention and data quality. The ODC is currently performing a natural history study of patients with GM1 funded by us.

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Compassionate Use Statement

Passage Bio is dedicated to developing transformative gene therapies for patients with severe, life threatening, rare neurologic disorders. We approach our work every day with great urgency and commitment to the patients and families who suffer from these catastrophic diseases for which there are few or no treatment options. 

Our commitment to clinical research

To ensure that we meet our responsibility to deliver safe and effective treatments to patients, we are collaborating with scientists, physicians, patients and health authorities around the world to conduct rigorous clinical trials. We believe that it is through clinical trials and regulatory approval that we can ultimately ensure safe and timely access to our medicines for all patients and families suffering from these devastating diseases.

Review process for Expanded Access requests

We understand that there may be circumstances under which patients or caregivers may request treatment with our investigative therapies before they have been approved by regulatory agencies. This is sometimes called “expanded access”, or “compassionate use” or a “named patient program”, and we use the term Expanded Access to include all of these. While we do not anticipate providing for Expanded Access to investigational therapies prior to completion and analysis of our registrational clinical trials, we have developed the following process to evaluate such requests for Expanded Access:

  • Before one of our investigative therapies is approved by regulatory authorities, any requests for Expanded Access to such investigative therapy received by Passage Bio will be referred to study investigators to determine a patient’s eligibility for one of our ongoing clinical studies with that product candidate.
  • If a patient does not qualify for one of our ongoing clinical studies, Passage Bio will direct such requests to patient organizations, medical professionals and/or scientists who may be able to identify other clinical studies or approved treatment options.
  • Once we have completed and analyzed data from our registrational trials, we will consider requests for Expanded Access on a case-by-case basis. Requests for Expanded Access will be considered based on a number of factors, including the following factors:
    • Based on available scientific data, there is a reasonable expectation that the mechanism of action and/or biological activity of the investigational therapy will provide therapeutic benefit to the individual patient’s disease;
    • Available safety and efficacy data for the investigational therapy and the potential benefits and risks for the individual patient;
    • The availability of alternative treatment options;
    • Compliance with legal and health authority requirements, including institutional review board (IRB) approvals;
    • Potential compromise of related clinical trials or regulatory pathways.
    • Feasibility considerations including but not limited to the availability of the investigational therapy supply, and the availability of expert clinical oversight to ensure the safe administration of investigational therapy and adequate clinical follow up.

Making a Request for Expanded Access

We are committed to evaluating all requests for Expanded Access in a fair and equitable manner. Requests for access to a Passage Bio investigational therapy must be made voluntarily by the patient’s treating physician. Physicians seeking Expanded Access for patients with no alternative treatment options should submit their requests to

Passage Bio will acknowledge requests from licensed physicians, generally within 10 business days of receipt and a decision will be made and communicated thereafter. Passage Bio retains full discretion to determine whether to grant requests for Expanded Access, and requests for access may not be granted, even when eligibility criteria are met.

Expanded Access to investigational therapies must comply with the applicable country-specific laws and regulations, including importation requirements, approvals from applicable regulatory bodies, and by an appropriate Institutional Review Board or Ethics Committee. Passage Bio may require agreements and/or certifications executed by the treating physician and other information to be provided prior to final approval of any request.

Patients and caregivers seeking information about access to our investigational therapies, either through a clinical trial or through Expanded Access, should consult with their treating physician.

Should changes be made to our Expanded Access programs for our investigational therapies, we will update this website.

Passage Bio may revise this Policy at any time as provided by the 21st Century Cures Act.