FDA Issues Complete Response Letter to Abeona’s Cell Therapy for Rare Skin Disease

1. The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Abeona Therapeutics regarding its cell therapy for a rare skin disease.
2. The cell therapy, known as EB-101, is being developed to treat patients with a severe form of the genetic skin disorder Epidermolysis Bullosa (EB).
3. The CRL indicates that the FDA has completed its review of the Biologics License Application (BLA) for EB-101 and has determined that it cannot approve the application in its current form.
4. The FDA's concerns primarily relate to manufacturing and quality control issues, as well as the need for additional clinical data to support the therapy's safety and efficacy.
5. Abeona Therapeutics has stated that it is committed to working closely with the FDA to address the agency's concerns and to advance the development of EB-101 as a potential treatment for patients with EB.
6. EB is a rare and debilitating genetic disorder that affects the skin and mucous membranes, causing painful blisters and wounds that can lead to serious complications, including infections, scarring, and even cancer.
7. There is currently no cure for EB, and treatment options are limited, making the development of new therapies like EB-101 critically important for patients and their families.