Fibrocell Receives FDA Regenerative Medicine Advanced Therapy Designation for FCX-007 Gene Therapy

Fibrocell Science (NASDAQ:FCSC) has announced that the US Food and Drug Administration (FDA) has granted the company Regenerative Medicine Advanced Therapy designation for its FCX-007 gene therapy to treat recessive dystrophic epidermolysis bullosa (RDEB).

As quoted in the press release:

Created under the 21st Century Cures Act, RMAT offers sponsors of cell and gene therapies eligibility for accelerated development and review of their product if it is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence showing it has the potential to address unmet medical needs. This designation makes a product eligible for the same actions to expedite the development and review of a marketing application that are available to drugs that receive Breakthrough Therapy designation, including earlier and more frequent meetings with the FDA and potential eligibility for Priority Review and Accelerated Approval.

“We are pleased that the FDA has granted RMAT designation to FCX-007, which we believe offers the potential to address an unmet medical need of RDEB patients,” said John Maslowski, President and Chief Executive Officer of Fibrocell. “We recognize this important designation has the potential to accelerate development and review of FCX-007, and we look forward to working closely with the FDA as our program advances into a Phase 3 clinical trial.”

The Company expects to initiate the Phase 3 clinical trial for FCX-007 in the second quarter of 2019. Fibrocell projects enrollment and dosing of Phase 3 patients will be completed in the third quarter of 2020 and data collection for the primary endpoint will be completed in the fourth quarter of 2020. If the Phase 3 clinical trial is successful and completed within the projected timeframe, Fibrocell expects to file a Biologics License Application (BLA) for FCX-007 in 2021.

Click here to read the full press release.