The FDA has granted Breakthrough Therapy Designation status for the EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa.
The recent designation was based on data from the Phase 1/2 EB-101 clinical trial. The EB-101 product, which is developed by Abeona Therapeutics Inc, is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s keratinocytes. Participants with non-healing chronic wounds received EB-101 and were assessed for wound healing at predefined time points. Results from the trial demonstrated significant wound healing (greater than 50% healed) in treated wounds for over 2 years.
The Phase 3 clinical trial for EB-101 is slated to begin in early 2018.
Breakthrough Therapy designation can expedite the development and review of drugs for serious or life-threatening conditions. The EB-101 program has already received Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency.
“EB-101 is an autologous gene-corrected therapeutic approach that utilizes a patient’s own cells, genetically engineering them to produce the correct version of collagen which helps hold skin onto the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” said Timothy J. Miller, PhD, Abeona president and chief executive officer. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal Phase 3 trial as we advance EB-101 for patients with this debilitating disease.”
–Julie Gould
The FDA has granted Breakthrough Therapy Designation status for the EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa.
The recent designation was based on data from the Phase 1/2 EB-101 clinical trial. The EB-101 product, which is developed by Abeona Therapeutics Inc, is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s keratinocytes. Participants with non-healing chronic wounds received EB-101 and were assessed for wound healing at predefined time points. Results from the trial demonstrated significant wound healing (greater than 50% healed) in treated wounds for over 2 years.
The Phase 3 clinical trial for EB-101 is slated to begin in early 2018.
Breakthrough Therapy designation can expedite the development and review of drugs for serious or life-threatening conditions. The EB-101 program has already received Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency.
“EB-101 is an autologous gene-corrected therapeutic approach that utilizes a patient’s own cells, genetically engineering them to produce the correct version of collagen which helps hold skin onto the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” said Timothy J. Miller, PhD, Abeona president and chief executive officer. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal Phase 3 trial as we advance EB-101 for patients with this debilitating disease.”
–Julie Gould
The FDA has granted Breakthrough Therapy Designation status for the EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa.
The recent designation was based on data from the Phase 1/2 EB-101 clinical trial. The EB-101 product, which is developed by Abeona Therapeutics Inc, is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s keratinocytes. Participants with non-healing chronic wounds received EB-101 and were assessed for wound healing at predefined time points. Results from the trial demonstrated significant wound healing (greater than 50% healed) in treated wounds for over 2 years.
The Phase 3 clinical trial for EB-101 is slated to begin in early 2018.
Breakthrough Therapy designation can expedite the development and review of drugs for serious or life-threatening conditions. The EB-101 program has already received Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency.
“EB-101 is an autologous gene-corrected therapeutic approach that utilizes a patient’s own cells, genetically engineering them to produce the correct version of collagen which helps hold skin onto the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” said Timothy J. Miller, PhD, Abeona president and chief executive officer. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal Phase 3 trial as we advance EB-101 for patients with this debilitating disease.”
–Julie Gould
