At Percheron our science goes beyond the development of novel therapies, and we are committed to engaging with and supporting rare and orphan disease communities.
Compassionate Use and Expanded Access Policy
At Percheron Therapeutics, we are committed to developing innovative therapies for rare diseases with significant unmet medical need. Our focus is on advancing cutting-edge treatments for patients who currently have limited or no effective options. Clinical trials play a critical role in the development of investigational therapies, providing the essential data needed to establish safety and efficacy for regulatory approval.
Clinical Trials and Investigational Treatments
Clinical trials are the primary and preferred way for patients to access investigational therapies. These trials are designed to generate the rigorous evidence required by regulatory authorities to evaluate whether a treatment can be made widely available to patients. We encourage patients and healthcare providers to explore ongoing clinical trials as the primary option for accessing investigational treatments. More information about clinical trials of Percheron Therapeutics’ investigational product ATL1102 (avicursen) can be found on clinicaltrials.gov, as well as on www.anzctr.org.au and other regulatory and clinical trial databases.
Future Considerations for Compassionate Use and Expanded Access
At this time, Percheron Therapeutics is not offering compassionate use, also known as expanded access or pre-approval access, for any of its investigational products. Our current focus remains on clinical trial participation to ensure that the data needed for regulatory approval are collected efficiently and robustly. However, we do make an attempt to offer short term continued access to avicursen after participation in a clinical trial ends, if deemed appropriate by the participant’s investigator and treating physician.
We recognize that there may be situations in the future where compassionate use could become an option for patients who have no other viable treatment alternatives and are unable to participate in clinical trials. Should Percheron Therapeutics decide to offer compassionate use in the future, we will consult with healthcare providers, regulatory authorities, and patient advocacy groups to ensure that any program we develop aligns with the highest ethical and scientific standards.
Working with Patient Communities
The groups below work to raise awareness of Duchenne muscular dystrophy in Australia and overseas and many of them are founded by parents of children with Duchenne muscular dystrophy within the community. These organisations assist in connecting patients and families to education, research, and support services.
