Expanded Access Policy
Benitec Biopharma, Inc. (“Benitec”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines. Benitec is focused on the development of its investigational genetic medicine, BB-301, for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) related moderate dysphagia. In conjunction with our ongoing work with key members of the clinical research community, the OPMD patient community and regulatory agencies, we are conducting clinical trials to assess the safety, tolerability, and efficacy of BB-301. The clinical safety and efficacy results from these trials could, potentially, support approval from regulatory authorities, which would allow broader access to BB-301.
Expanded access (sometimes referred to as “compassionate use”) is a potential pathway for a patient with a serious disease or immediately life-threatening condition to gain access to an investigational drug for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
At this time, access to BB-301 is possible only through participation in our clinical trials until there are sufficient clinical results to demonstrate safety, tolerability, and efficacy which can be confirmed by regulatory authorities. Clinical trials are conducted in specific patient populations and carefully monitored and regulated to allow for the assessment of safety and to determine risks and potential benefits of investigational treatments, including genetic medicines. Expanded access outside of a clinical trial may interfere with the conduct of ongoing clinical trials and delay access to BB-301 for the broader patient community. Our ultimate goal is to provide global access to BB-301 to patients around the world. As a result of these considerations, we are not accepting expanded access requests at this time. However, Benitec reserves the right to change this policy at its discretion and may offer study material for compassionate use in the future once additional data are available.
You can find additional information about Benitec’s current ongoing clinical trial by accessing https://clinicaltrials.gov, ClinicalTrials.gov Identifier: NCT06185673.
For additional information about this policy, please contact info@benitec.com.