09.25.18
Fibrocell Science announced that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) has awarded a $1.4 million clinical trial research grant for Fibrocell’s continued clinical development of FCX-007, the Company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a devastating, rare skin blistering disease with high mortality.
“We are delighted with the FDA’s recognition to support the ongoing progress of our clinical trials of FCX-007,” said John Maslowski, president and chief executive officer of Fibrocell. “With no FDA approved therapies available, this grant further validates the significant opportunity of FCX-007’s potential to relieve the pain and suffering from the debilitating, chronic blisters and open wounds of RDEB and offer hope to patients and their families.”
Fibrocell’s $1.4 million grant, which will be distributed over the next four years, was awarded by the FDA through the OOPD’s Orphan Products Clinical Trials Grants Program. This program supports the clinical development of products for use in rare diseases or conditions for which “no current therapy exists or where the proposed product will be superior to the existing therapy.”
FCX-007 is currently being evaluated in the Phase 2 portion of a Phase 1/2 clinical trial for the treatment of RDEB. Six patients ages seven and older are targeted to be treated with FCX-007 in the Phase 2 portion of the clinical trial. Fibrocell expects to report an interim data analysis for FCX-007 and provide a clinical trial update from Phase 1 patients and available data from Phase 2 patients in the first quarter of 2019.
The FDA has granted Orphan Drug Designation for the treatment of dystrophic epidermolysis bullosa, including RDEB, Rare Pediatric Disease Designation for the treatment of RDEB and Fast Track Designation for the treatment of RDEB to FCX-007.
“We are delighted with the FDA’s recognition to support the ongoing progress of our clinical trials of FCX-007,” said John Maslowski, president and chief executive officer of Fibrocell. “With no FDA approved therapies available, this grant further validates the significant opportunity of FCX-007’s potential to relieve the pain and suffering from the debilitating, chronic blisters and open wounds of RDEB and offer hope to patients and their families.”
Fibrocell’s $1.4 million grant, which will be distributed over the next four years, was awarded by the FDA through the OOPD’s Orphan Products Clinical Trials Grants Program. This program supports the clinical development of products for use in rare diseases or conditions for which “no current therapy exists or where the proposed product will be superior to the existing therapy.”
FCX-007 is currently being evaluated in the Phase 2 portion of a Phase 1/2 clinical trial for the treatment of RDEB. Six patients ages seven and older are targeted to be treated with FCX-007 in the Phase 2 portion of the clinical trial. Fibrocell expects to report an interim data analysis for FCX-007 and provide a clinical trial update from Phase 1 patients and available data from Phase 2 patients in the first quarter of 2019.
The FDA has granted Orphan Drug Designation for the treatment of dystrophic epidermolysis bullosa, including RDEB, Rare Pediatric Disease Designation for the treatment of RDEB and Fast Track Designation for the treatment of RDEB to FCX-007.
