Fibrocell received $1.4 million from the U.S. Food and Drug Administration’s Office of Orphan Products Development (OOPD) to support the company’s ongoing clinical trials testing its FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
In collaboration with Precigen (a wholly owned subsidiary of Intrexon), Fibrocell developed the experimental gene therapy FCX-007 to correct the cause of RDEB — a defect in the gene Col7 that leads to the production of a faulty type VII collagen protein. Collagen is a protein that binds skin layers, and a defective type VII collagen protein results in frequent blistering and fragile skin.
The new funding was awarded by the OOPD’s Orphan Products Clinical Trials Grants Program, and is to be distributed over the next four years.
According to the FDA press announcement about these awards, “the grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to the treatment of rare diseases.
“Grant applications were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, which included representatives from academia, the National Institutes of Health and the FDA,” the agency stated.
The grant awarded to Fibrocell will support the ongoing Phase 1/2 trial (NCT02810951) assessing the safety of FCX-007 and its effectiveness to promote wound healing in RDEB patients. The trial, which lasts 52 weeks, will evaluate the patient’s response to FCX-007 at multiple time points.
Encouraging interim results of the Phase 1 part of the trial were recently reported. Four adult RDEB patients (ages 20-37) received tailored FCX-007 intradermal injections. Among the four patients, a total of seven wounds were treated.
At the interim analysis at four and 12 weeks after treatment, more than 50% healing was seen in seven (100%) and six (86%) of the wounds treated, respectively.
For the Phase 2 part of the trial, Fibrocell intends to treat six younger patients (age 7 and older).
The company expects to report initial data on these trials in the first quarter of 2019.
“We are delighted with the FDA’s recognition to support the ongoing progress of our clinical trials of FCX-007,” John Maslowski, president and CEO of Fibrocell, said in a press release.
“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 offers hope to patients and their families,” Maslowski added.
FCX-007 received FDA fast-track designation for the treatment of RDEB in early 2017, in addition to orphan drug status.