The deal places Scioderm’s lead product candidate Zorblisa, a topical cream for the treatment of epidermolysis bullosa (EB), into Amicus’ rare and orphan disease pipeline.
In a prepared statement, Amicus CEO John F. Crowley, who serves on the Scioderm board of directors, said Zorblisa will bring relief to patients living with EB.
“EB is a disorder that is utterly devastating and painful as it causes extremely fragile skin that blisters and tears from minor friction or trauma. In many children, it leads to severe complications and a very early death,” said Crowley. “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.”
Zorblisa is now in phase III trials. In 2013, it received a Breakthrough Therapy designation from the FDA after early trials showed its ability to help heal wounds across the spectrum of EB subtypes.
Amicus estimated that Zorblisa will generate $1 billion in annual revenues. Existing treatments for EB have a monthly cost of between $10,000 and $15,000. The company said most treatments are palliative in nature and consist primarily of “bandaging, treating the open wounds to prevent infection and trying to manage patients’ pain.” According to a company estimate, there are between 30,000 and 40,000 people “in major markets” who have been diagnosed with EB.
The acquisition of Scioderm fits into Amicus’ plan of bringing more first-in-class, rare-disease drugs into the company. Crowley has said he first proposed the deal to Scioderm earlier this year.