FDA Grants Orphan Drug Designation to Fibrocell’s FCX-013, a Gene Therapy for Localized Scleroderma

FDA Grants Orphan Drug Designation to Fibrocell’s FCX-013, a Gene Therapy for Localized Scleroderma
0
(0)

The U.S. Food and Drug Administration (FDA) has designated a potential gene therapy for localized scleroderma, FCX-013 by  Fibrocell Science, an orphan drug to help advance its development and testing.

Fibroblasts, the basis of Fibrocell’s personalized cell and gene therapy platform, are the most common cell in skin and connective tissue, and responsible for synthesizing extracellular matrix proteins, including collagen, which provides structure and support.

FCX-013 is an autologous dermal fibroblast transduced with a gene that encodes a protein able to regulate collagen accumulation at the site of the localized disease (through the breakdown of excess collagen). It incorporates Intrexon’s proprietary RheoSwitch Therapeutic System, designed to allow researchers to control future expression of the protein once fibrosis has been resolved. FCX-013 is currently in preclinical development for the treatment of linear scleroderma.

“Achieving orphan drug designation for FCX-013 is an important regulatory milestone for us,” said David Pernock, chairman and chief executive officer of Fibrocell, in a recent press release. “We recently reported the successful completion of a proof-of-concept study in which FCX-013 reduced the dermal thickness of fibrotic tissue in a scleroderma rodent model. Based on these results, we advanced FCX-013 into pre-clinical dose ranging and toxicology/biodistribution studies for product optimization. We expect to submit an Investigational New Drug application for FCX-013 to the FDA in 2017.”

FCX-013 is the company’s second gene-therapy candidate receiving orphan drug designation, the other being FCX-007. The FDA also recently approved the start of a Phase 1/2 study of FCX-007 as a potential treatment of recessive dystrophic epidermolysis bullosa.

Localized scleroderma is a chronic autoimmune disease characterized by thickening of the skin and connective tissue, and can be debilitating and painful.

Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
×
Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
Latest Posts
  • preclinical study
  • EHP-101 Emerald
  • PAH letters
  • Actemra trial enrollment ends

How useful was this post?

Click on a star to rate it!

Average rating 0 / 5. Vote count: 0

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?