Amicus Therapeutics of Cranbury, New Jersey, USA to take over a Durham-based company called Scioderm for $229 million. The company's unique treatment for blisters and lesions in patients with epidermolysis bullosa (EB) is currently under the phase III trial stage.
"We believe we are well-positioned to rapidly complete the clinical development of Zorblisa and to make Zorblisa commercially available for all EB patients as quickly as possible," said John F. Crowley, chairman and chief executive officer of Amicus and board member of Scioderm.
EB affects around 30,000 to 40,000 people in the United States and 400,000 people worldwide. Therefore Amicus estimates that the treatment may contribute to a $1 billion-plus global market opportunity.
EB is a rare debilitating disease characterized by fragile skin that blisters and tears from minor friction or trauma. Young EB patients are often known as "butterfly children" because their skin is delicate like the wings of a butterfly. The current standard of care is palliative treatments that cost $10,000 to $15,000 per month and mainly consist of bandaging, treating the open wounds to prevent infection.
"This combination of Amicus and Scioderm is a major win for EB patients. With the added resources and expertise that Amicus provides for the Zorblisa program, we are more confident than ever in our potential for success and our ability to deliver significant benefits to patients and families living with the devastating effects of EB," said Robert Ryan, Chief Executive Officer of Scioderm.
Zorblisa is the first "breakthrough therapy" recognized by the USFDA for EB. It is the only treatment to show significant effects on wounds due to EB across all its major subtypes during its phase II trial.
"Amicus is a champion of the rare disease community that, together with Scioderm, understands our sense of urgency to see a treatment approved for EB. The EB community will be well-served by the experience and broad, global capabilities that Amicus adds to Scioderm," said Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America.