FDA Grant Supports Recessive Dystrophic Epidermolysis Bullosa Gene Therapy Study


Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.

The FDA has granted an orphan products development grant to Castle Creek Biosciences to support the phase 3 DeFi-RDEB study (NCT04213261) of the gene therapy dabocemagene autoficel (FCX-007, D-Fi) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).1

“We are honored to be awarded this highly competitive research grant for the ongoing clinical development of FCX-007, which has the potential to transform the lives of people suffering from dystrophic epidermolysis bullosa (DEB),” principal investigator Mary Spellman, MD, chief medical officer, Castle Creek Biosciences, said in a statement.1

The $1.825 million research grant will be distributed over four years. The grant will support a meaningful portion of the clinical development of the investigational gene therapy dabocemagene autoficel. The gene therapy is designed to correct the functional type VII collagen protein (COL7) deficiency in patients with DEB and consists of autologous, genetically engineered, dermal fibroblasts with a lentiviral vector to deliver the COL7A1 gene. The therapy is locally injected into the papillary dermis of persistent and non-healing DEB recurrent wounds where the COL7 protein can support the formation of anchoring fibrils in the skin.

The FDA previously granted orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations to dabocemagene autoficel for the treatment of RDEB.

READ MORE: Gene Therapy for Congenital Hearing Loss Granted Orphan Drug, Rare Pediatric Disease Designations

‘We are committed to advancing our study of FCX-007 to develop a durable personalized therapy for the localized treatment of chronic wounds due to RDEB and will continue to work closely with the FDA as our program progresses,” Spellman added.1 

The multi-center, randomized, controlled, open-label DeFi-RDEB study is evaluating the safety and efficacy of dabocemagene autoficel in up to 24 patients with RDEB. Participants’ wounds are randomized to either receive the therapy or remain untreated. The trial is currently recruiting at 5 sites, including Stanford University, Children’s Hospital Colorado, Dell Children’s Medical Group, Solutions Through Advanced Research, Inc, and Mayo Clinic. 

DeFi-RDEB is primarily assessing efficacy as measured by complete wound closure of the first wound pair at week 24. Secondary efficacy outcomes include complete wound closure of the first pair at week 12, complete wound closure of all pairs at week 24, and complete wound closure of all pairs at week 12. 

“Our late-stage Phase 3 trial of D-Fi continues to progress for the localized treatment of RDEB, a devastating condition for too many families who currently do not have options beyond palliative care,” John Maslowski, former chief executive officer and current chief scientific advisor and chairman, scientific advisory board, Castle Creek Biosciences, said in a previous statement.2

Castle Creek Biosciences also recently announced a research collaboration with Mayo Clinic to expand its gene therapy platform and develop candidates to treat osteogenesis imperfecta and classical Ehlers-Danlos syndrome.3 The research will be led by principal investigator David R. Deyle, MD, geneticist, department of medical genetics, Mayo Clinic.

“We are honored to be working with Dr. Deyle and his highly regarded research team at Mayo Clinic to identify and evaluate gene therapy candidates that hold promise for treating debilitating, rare connective tissue disorders with high unmet medical needs,” Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, previously said in a statement.3 “We expect this initiative will be the first of multiple Castle Creek strategic collaborations with leading medical research institutions that have the potential to expand our innovative gene therapy discoveries for rare diseases and offer hope to underserved patient communities impacted by these devastating conditions.” 

1. Castle Creek Biosciences awarded FDA orphan products development grant to support DeFi-RDEB, a pivotal phase 3 study of FCX-007 investigational gene therapy for recessive dystrophic epidermolysis bullosa. News release. Castle Creek Bioscience. October 21, 2021. https://castlecreekbio.com/castle-creek-biosciences-awarded-fda-orphan-products-development-grant-to-support-defi-rdeb-a-pivotal-phase-3-study-of-fcx-007-investigational-gene-therapy-for-recessive-dystrophic-epidermolysis-bull/
2. Castle Creek Biosciences announces first patient dosed in DeFi-RDEB phase 3 clinical trial of D-Fi gene therapy for recessive dystrophic epidermolysis bullosa. News release. Castle Creek Biosciences. October 28, 2020. Accessed October 27, 2021. https://castlecreekbio.com/castle-creek-biosciences-announces-first-patient-dosed-in-phase-3-clinical-trial-of-d-fi/
3. Castle Creek Biosciences expands its innovative gene therapy platform for rare genetic connective tissue disorders through research collaboration with Mayo Clinic. News release. Castle Creek Biosciences. September 29, 2021. https://castlecreekbio.com/castle-creek-biosciences-expands-its-innovative-gene-therapy-platform-for-rare-genetic-connective-tissue-disorders-through-research-collaboration-with-mayo-clinic/
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