SD-101 is a topical cream that has demonstrated potential to provide improvement in treating the severe skin disruptions seen in patients across all EB subtypes.

Paradigm Therapeutics Inc. now has the worldwide rights of SD-101, a topical whole-body treatment for all subtypes of Epidermolysis Bullosa (EB), from Amicus Therapeutics. 

Terms of the deal have not been disclosed.

SD-101 received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) covering the broad treatment of EB. In addition to the Rare Pediatric Disease designation, SD-101 was granted Orphan Drug designation from the FDA and the European Commission (EC) and was the first EB treatment to receive the FDA's Breakthrough Therapy designation.

SD-101 is a topical cream that has demonstrated potential to provide improvement in treating the severe skin disruptions seen in patients across all EB subtypes. Phase II and Phase III studies were conducted in children and adults with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB. A total of 217 EB patients were included in these studies. SD-101 was applied topically over the entire body daily in adults and children as young as 21 days old for a period of three months. The primary outcome measurement was assessment of target wound closure, in addition to secondary measurements including reduction in body surface area (BSA) coverage of blisters and wounds. 

"We are excited about the opportunity to continue the development of SD-101 and to discuss the current data with the FDA to expedite a path forward towards registration of this therapy for patients with all EB subtypes," says Robert Ryan., Ph.D., Chief Executive Officer of Paradigm Therapeutics, in a news release. "There is a tremendous need for novel treatment options for treating the debilitating skin effects in patients with Epidermolysis Bullosa. The Paradigm Therapeutics team will be working passionately to provide patients with a new therapeutic alternative, in this disease with no effective whole-body treatments."

Dr. Ryan adds, "Epidermolysis Bullosa is a devastating disease. We believe the clinical data show beneficial effects of the use of SD-101 across the range of EB patients, and strongly support progression towards registration. Results from the completed Phase II and Phase III trials indicate that SD-101 is a product candidate for whole body treatment of the lesions and wounds on skin in pediatric and adult patients with EB, who have no other whole body treatment alternatives. Results also demonstrated that SD-101 has a favorable safety profile."