Paradigm Acquires Late-Stage Therapy for Treatment of All Subtypes of Epidermolysis Bullosa

Rare Daly Staff

Paradigm Therapeutics acquired from Amicus Therapeutics worldwide rights for SD-101, a topical whole-body treatment for all subtypes of epidermolysis bullosa.

Financial terms of the deal have not been disclosed.

Epidermolysis bullosa (EB) is a rare, devastating genetic disorder causing very fragile skin that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies to treat the whole body of patients with EB, which affects many children and adults throughout their lifetimes. The primary standard of care consists of pain management, and cleaning and bandaging of open wounds to prevent infection.  EB is a chronic disease, potentially disfiguring, and in some cases fatal. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma.

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 2 and phase 3 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 coverage of blisters and wounds.

SD-101 received Rare Pediatric Disease designation from the U.S. Food and Drug Administration 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 and was the first EB treatment to receive the FDA’s Breakthrough Therapy designation.

“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.,” said Robert Ryan, CEO of Paradigm Therapeutics. “Results from the completed phase 2 and phase 3 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.”

Photo: Robert Ryan, CEO of Paradigm Therapeutics