One month after Fibrocell submitted an Investigational New Drug application with the U.S. FDA for FCX-013, the application for the gene therapy to treat scleroderma was granted allowance.
One month after Fibrocell Science, Inc. submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for FCX-013, the company has announced that the application for the gene therapy candidate to treat moderate to severe localized scleroderma was granted allowance.
Fibrocell expects to initiate enrollment for an open label, single arm Phase 1/2 clinical trial in the third quarter of 2018.
“We are pleased the FDA has granted allowance of our IND for FCX-013 to begin clinical trials for the treatment of moderate to severe localized scleroderma, offering patients the potential for relief from this chronic, painful and debilitating disorder,” said John Maslowski, President and Chief Executive Officer of Fibrocell in a press release. “With no FDA-approved therapies available, we look forward to advancing FCX-013 into the clinic.”
Localized scleroderma, a chronic autoimmune disease, is characterized by a thickening of the skin as the result of excessive collagen deposits. FCX-013 is being designed to express matrix metalloproteinase 1 (MMP-1), the protein responsible for breaking down superfluous collagen.
FCX-013 is designed to be injected under the skin as the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation.
The FDA has previously granted Orphan Drug Designation to FCX-013 for the treatment of localized scleroderma, as well as a Rare Pediatric Disease Designation for the treatment of moderate to severe localized scleroderma.
Fibrocell hopes to enroll 10 patients with any type of localized scleroderma into an open label, single arm Phase 1/2 clinical trial that is expected to begin in the third quarter of 2018. The primary objective will evaluate the safety of the drug, while secondary endpoints include a number of fibrosis assessments, like histology, skin scores, ultrasound and additional measurements of targeted sclerotic lesions and control sites at various time points up to 16 weeks post-administration.
The Phase 1 portion will enroll adult patients, and dosing for the first three will be staggered prior to dosing the rest of the trial’s population. The Phase 2 portion will include pediatric patients, and will begin after safety and activity data from the Phase 1 has been submitted to and approved by the FDA and the trial’s Data Safety Monitoring Board.
“The impact of localized scleroderma on patients, particularly children, can be devastating—affecting growth and mobility of their affected limbs,” said Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine. “There are no approved therapies for localized scleroderma. Current treatments are aimed at impacting inflammation, but few options exist to treat the excessive collagen deposition in the skin and soft tissue, which may produce pain and limitation in motion and growth. With FCX-013, the goal is to bring relief to patients by targeting the abnormal collagen metabolism to improve skin function.”