Breakthrough Therapy Designation Granted to SD-101 for Treatment of Epidermolysis Bullosa

Paradigm Therapeutics Inc. recently announced the acquisition of worldwide rights of SD-101, a topical whole-body treatment for all subtypes of epidermolysis bullosa (EB), from Amicus Therapeutics.¹ SD-101 received Rare Pediatric Disease designation from the US Food and Drug Administration (FDA) encompassing the broad category of EB.

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SD-101 is a topical cream that has shown potential for the treatment of all subtypes of EB. In phase II and phase III trials of 217 children and adults with either simplex, recessive dystrophic (RDEB), or junctional EB, the cream was applied all over the body. The cream was used on patients as young as 21 days old for 3 months. The primary outcome measurement was evaluation of target wound closure. Secondary measures included reduction in body surface area of blisters and wounds.

SD-101 received 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.

"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," said Robert Ryan, PhD, chief executive officer of Paradigm Therapeutics in a press 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."

Ryan said, “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."

Paradigm Therapeutics is a clinical-stage pharmaceutical company with a focus on developing innovative therapies for the treatmentof rare diseases.

Reference

1. Paradigm Therapeutics acquires late stage “Breakthrough Therapy” designated therapy for treatment of all subtypes of epidermolysis bullosa. News release. August 22, 2023. Accessed August 22, 2023. https://prnmedia.prnewswire.com/news-releases/paradigm-therapeutics-acquires-late-stage-breakthrough-therapy-designated-therapy-for-treatment-of-all-subtypes-of-epidermolysis-bullosa-eb-301903917.html