At GEMMABio, we are prioritizing research, innovation, and accelerated global access to life-changing advanced therapies for those living with rare diseases. Above all, we are deeply committed to improving patient lives by delivering the most safe and effective treatments to rare disease patients in need.
The U.S. FDA’s expanded access program – also known in various other jurisdictions as compassionate use, early access, individual or named patient program, special access, unlicensed medicines, or market access – allows eligible patients to request access to investigational medical products without participating in a clinical trial, in qualifying circumstances for serious diseases where there is no alternative treatment available.
At this time, GEMMABio does not currently offer any expanded access programs for our investigational therapeutics. GEMMABio’s manufacturing capacity and drug supply for investigational drugs are dedicated entirely to the scope of each clinical trial.
To preserve our product supply and ensure patient safety and the scientific integrity of our programs, we believe that access to our investigational therapeutics should be preceded by rigorous evaluation of the highest standards for safety, efficacy, dose optimization and risk/benefit analysis. Should we make expanded access to our investigational therapeutics available in the future, we will update this policy with a procedure for making requests and our evaluation criteria.
You and your health care provider may learn more about GEMMABio’s clinical trials by visiting www.clinicaltrials.gov and searching for GEMMA Biotherapeutics. If you are a health care provider who is interested in learning more about our investigational therapeutics, or a physician with questions about participation in one of our clinical trials, please submit a request to clinical_studies@gemmabiotx.com. We expect to acknowledge questions as soon as possible, usually within 5 business days of receipt.
We remain dedicated to advancing our clinical programs efficiently and responsibly and will continue to evaluate expanded access opportunities as our efforts advance. Please check this page periodically as we reserve the right to update this policy at any time.
