Posted by Michael Wonder on 30 Jan 2018
Abeona receives FDA regenerative medicine advanced therapy designation for EB-101 gene therapy in epidermolysis bullosa
29 January 2018 - First gene therapy RMAT designation for epidermolysis bullosa.
Abeona Therapeutics announced today that the US FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to EB-101, the Company’s gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa.
The Company continues to engage the FDA on its pivotal Phase 3 clinical trial design, and will provide an update on the program in the coming months. Abeona’s EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) for the treatment of the underlying disease in recessive dystrophic epidermolysis bullosa. The EB-101 program has been granted breakthrough therapy, orphan drug and rare paediatric disease designations from the US FDA and orphan drug Designation from the EMA.
