FDA Advises Fibrocell on Phase 3 Trial Design for FCX-007, Potential RDEB Therapy
Fibrocell Science has completed a type C meeting with the U.S. Food and Drug Administration in which they discussed the design of a Phase 3 clinical trial to assess the efficacy of FCX-007, a gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
During the meeting, the FDA was able to provide advice and guidance on several design aspects of a future Phase 3 trial. The FDA also counseled Fibrocell on the chemistry, manufacturing, and control requirements for the proposed trial, and offered guidance for a prospective biologics license application for FCX-007.
Based on the FDA’s feedback, Fibrocell will likely submit the edited protocol in the fourth quarter of 2018, and will give further information on the clinical trial design once it is finalized.
Fibrocell Science is developing FCX-007 in partnership with Precigen, a wholly owned subsidiary of Intrexon. FCX-007 is a gene therapy designed to correct the cause of RDEB — a defect in the Col7 gene that leads to the production of a faulty type VII collagen protein, which results in frequent blistering and fragile skin.
As FCX-007 treatment can provide large amounts of COL7 directly to the affected regions, this therapeutic has the potential to treat the underlying cause of RDEB while at the same time avoiding affecting other regions of the body.
“We appreciate the valuable and detailed guidance furnished by the FDA on the design of the proposed Phase 3 clinical trial and advice on CMC requirements,” John Maslowski, president and CEO of Fibrocell, said in a press release. “We look forward to finalizing the Phase 3 protocol design, and plan to initiate the Phase 3 clinical trial in the first half of 2019.”
Fibrocell’s meeting with the FDA was based on the latest results from an ongoing Phase 1/2 trial of FCX-007 (NCT02810951). The objective of this study is to evaluate the safety and efficacy in regards to wound healing of FCX-007 intradermal (into-the-skin) injections in patients with RDEB.
As of now, Fibrocell has evaluated FCX-007 in five RDEB patients in the Phase 1/2 trial. The company plans to move forward with the Phase 2 part of this study, while submitting the Phase 3 protocol to the FDA at the same time.
The type C meeting with the FDA was also facilitated by the newest draft guidance from the agency focused on the field of gene therapy and epidermolysis bullosa. A draft guidance describes the FDA’s current thinking on recommended practices for drug and medical device manufacturers and their representatives.
FCX-007 has previously received orphan drug, rare pediatric disease, and fast track designations from the FDA.