Orphan Drugs: FDA Designations For Epidermolysis Bullosa
On June 10th, the FDA grants Orphan Drug Designation (ODD) to Fibrocell Technologies’ new investigational treatment for Dystrophic Epidermolysis Bullosa (DEB). EB is a group of genetic conditions causing one’s skin to be very delicate and to blister easily. A minor injury, scratching, or rubbing results in skin blisters and skin erosions. DEB is one of the major forms of EB and can vary from mild to severe. In the United States, there are approximately 2,800 – 5,600 patients. There is no treatment or cure for DEB. The rare disease is caused by mutations in the COL7A1 gene, which:
“provides instructions for making a protein used to assemble Type VII collagen. Collagens are molecules that give structure and strength to connective tissues, such as skin, tendons, and ligaments, throughout the body. Type VII collagen plays an important role in strengthening and stabilizing the skin”.
Fibrocell Technologies (Exton, Pennsylvania) is a subsidiary of Fibrocell Science, a biotechnology company that develops therapeutics using a person’s own cells to target localized treatment of rare and serious skin and connective tissue diseases – personalized medicine. Fibrocell Technologies’ platform involves human autologous fibroblast cell therapy that extracts a patient’s fibroblast cells, and then a gene is “transduced into an autologous fibroblast cell and administered into the patient to enable the production of the desired protein(s).”
FDA ODD Designation Database Record
|Generic Name:||autologous genetically modified human dermal fibroblasts|
|Orphan Designation:||Treatment of dystrophic epidermolysis bullosa|
|Orphan Designation Status:||Designated|
|FDA Orphan Approval Status:||Not FDA Approved for Orphan Indication|
|Sponsor:||Fibrocell Technologies, Inc. 405 Eagleview Blvd Exton, PA 19341 The sponsor address listed is the last reported by the sponsor to OOPD.|
Shire’s FDA ODD For DEB
In January 2013, Shire signs an agreement to acquire Lotus Tissue Repair that is developing the only protein replacement therapy being investigated for the treatment of DEB. In March 2014, the company receives FDA ODD for the treatment of DEB.
Please Note: “Pathologist with Microscope” courtesy of Bill Branson (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].
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