GC Biopharma is currently developing GC1130A, a potential enzyme replacement therapy for patients with Sanfilippo syndrome type A. We understand there may be interest in access to investigational product for patients outside of clinical trial. At this stage of clinical development, participation in a clinical study is the only way to gain access to GC Biopharma’s investigational product.
We are committed to supporting patients with Sanfilippo syndrome type A and their families by striving to obtain regulatory approval to provide access to as many patients as possible who are most likely to benefit. As we gather more clinical data on the safety and efficacy of GC1130A, we will carefully review and reconsider our Expanded Access Policy as needed to ensure it aligns with the best interests of patients.
If you have any questions about the GC Biopharma Expanded Access Policy, please contact GC Biopharma at ExpandedAccess@gccorp.com. GC Biopharma anticipates acknowledging receipt of any questions usually within 5 business days. Please do not send any personal health information to us.