Innovative Therapies
Expanded Access Policy 2018-01-04T09:14:09+00:00

Expanded Access Policy

Audentes is committed to developing and commercializing safe and effective gene therapy products for patients living with serious, life-threatening, rare diseases with limited or no treatment options. We appreciate the urgency of bringing these innovative products to patients, and this commitment drives our work each day. We believe in the importance of collaborating closely with patients, their families, patient advocacy organizations, physicians, researchers and regulatory authorities to help achieve this goal.

Our Approach to Clinical Development for Rare Disease

A robust and comprehensive clinical development program – designed with input from experts in the medical community, regulatory authorities and the patient community – is the optimal way to bring an approved therapy to the entire patient community.

Clinical trials are designed and implemented to gain an understanding of the safety and effectiveness of investigational new therapies in specific populations. Participation in clinical trials accepted by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), U.K. Medicines and Healthcare Products Regulatory Agency (MHRA), or other regulatory authorities, is the best way for patients to access investigational therapies for their diseases.

It is our hope and intention to quickly enroll eligible patients into clinical studies in order to demonstrate the safety and effectiveness of our investigational gene therapy products, and to rapidly obtain the global regulatory approvals needed to make these therapies widely available to the patients that need them around the world.

Expanded Access

We take the subject of expanded access (also known as “compassionate use” or “early access”) seriously, and have an internal team dedicated to this subject. We work in partnership with clinical experts in the medical field, and strive to obtain input from the patient advocacy communities whenever possible.

Per regional regulatory guidelines, expanded access programs must not interfere with ongoing or planned clinical trials designed to enable approval and access for the broader patient community. However, we recognize that in the rare disease setting and with diseases having a significant unmet medical need and limited or no available treatment options, it may be appropriate to offer expanded access in certain situations.

X-Linked Myotubular Myopathy (XLMTM)

Our priority is to demonstrate the safety and effectiveness of AT132, our gene therapy product for XLMTM, in order to obtain regulatory approval and make it available to all appropriate patients around the world as rapidly as possible. We do this by running a thoughtfully designed and robust clinical trial program.

If AT132 has demonstrated safety and effectiveness, and an optimal dose has been determined within the clinical trial, an expanded access program may be considered for certain children affected by XLMTM who are ineligible for the ASPIRO clinical trial.

Other Programs in Development

At this time, participation in clinical trials is the only way for patients to gain access to investigational therapies that are currently early in development. We recognize that not all patients will meet the eligibility requirements for these trials, and as more information and clinical data on the safety and efficacy of these investigational therapies become available, this position may change.

More Information

Treating physicians, patients and/or caregivers interested in learning more about Audentes’ investigational gene therapies currently undergoing clinical studies can find more information here. The linked webpages also include links to, which contains additional information about these studies.

For physicians or healthcare providers interested in more information, please contact medinfo(at) We anticipate acknowledging receipt of questions or request for more information within five (5) business days of receipt.

For parents or patients interested in general information, please contact Patient Advocacy at patientadvocacy(at)

General information on expanded access may be found at: