Compassionate Use

Neuren is committed to developing innovative medicines to treat serious, unmet medical needs for patients that suffer from rare neurodevelopmental disorders. We believe the best way to fulfill this responsibility is to conduct clinical trials to assess safety and effectiveness to support regulatory approval and commercialization so that our medicines may appropriately reach as many patients as possible.

We understand there may be interest in our investigational (pre-regulatory agency approval) medicines outside of our clinical trials through a compassionate use program, for example, Expanded Access in the United States. However, we believe the most appropriate way to access our investigational medicines is to participate in clinical trials regulated by the US Food and Drug Administration (FDA) and other regulatory agencies across the world and to partner with them for regulatory approval.

Therefore, at this time, we are unable to offer a compassionate use program without jeopardizing our ability to conduct the clinical trials needed to gain timely regulatory approval and provide patients wider availability and sustainable access.

Neuren complies with good clinical practices and takes steps to help ensure the quality and integrity of our clinical trials and to minimize risks to current research participants. For a list of clinical trials currently recruiting patients, please visit www.clinicaltrials.gov.