Amicus Therapeutics, a biotechnology company at the forefront of therapies for rare and orphan diseases, and Scioderm, Inc., a privately-held biopharmaceutical company focused on developing innovative therapies for treating diseases with high unmet need, have signed a definitive agreement under which Amicus will acquire 100 per cent of the capital stock of Scioderm, Inc.
Scioderm’s lead product candidate Zorblisa is a novel, late-stage, proprietary topical cream and potential first-to-market therapy for EB. Zorblisa has established positive proof-of-concept in phase 2 studies for the treatment of lesions in patients suffering with EB, and is currently being investigated in a phase 3 study to support global regulatory approvals. Zorblisa was one of the first products to receive FDA breakthrough therapy designation in 2013, and was the first-ever treatment in EB clinical studies to show significant benefit in wound closure across all major EB subtypes.
Amicus estimates that EB may represent a potential $1 billion+ global market opportunity based on third party market research. The current standard of care is palliative treatments which cost $10,000 to $15,000 per month, and mainly consist of bandaging, treating the open wounds to prevent infection and trying to manage patients’ pain. An estimated 30,000 to 40,000+ people are currently diagnosed with EB in major markets.