- Paradigm Therapeutics acquires late-stage therapy designated "Breakthrough Therapy" for the treatment of all subtypes of epidermolysis bullosa (EB)
'Topical therapy for the treatment of wounds and lesions on the entire surface of the skin'
MT PLEASANT, S.C., Aug. 22, 2023 /PRNewswire/ -- Paradigm Therapeutics Inc., a biopharmaceutical company, today announced the completion of its acquisition of the worldwide rights to SD-101, a topical whole-body treatment for all Epidermolysis Bullosa (EB) Subtypes, from Amicus Therapeutics. Terms of the agreement have not been disclosed.
SD-101 received rare pediatric disease designation from the US Food and Drug Administration (FDA) which covers the broad treatment of EB. In addition to the Rare Pediatric Disease designation, SD-101 received Orphan Drug designation from the FDA and the European Commission (EC) and was the first treatment for EB to receive Breakthrough Therapy designation from the FDA.
"We are excited about the opportunity to continue the development of SD-101 and discuss the current data with the FDA to accelerate the path toward registration of this therapy for patients with all EB subtypes," explained Robert Ryan, Ph.D. ., CEO of Paradigm Therapeutics. "There is a tremendous need for new treatment options to address the debilitating effects of the skin in patients with epidermolysis bullosa. The team at Paradigm Therapeutics will work passionately to provide patients with a new therapeutic alternative, in this disease with no effective treatments for the whole body".
Ryan added: "Epidermolysis bullosa is a devastating disease. We believe that the clinical data show beneficial effects of the use of SD-101 in the full range of EB patients and strongly support progression to registration. The Phase II results and Phase III trials indicate that SD-101 is a candidate product for the total body treatment of skin lesions and wounds in pediatric and adult EB patients, who have no other total body treatment alternatives. Results also demonstrated that SD-101 has a favorable safety profile."
About epidermolysis bullosa (EB)
Epidermolysis bullosa is a rare and devastating genetic disorder that causes very fragile skin leading 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 lives. The main standard of care consists of pain management and cleaning and dressing open wounds to prevent infection. EB is a chronic, potentially disfiguring, and in some cases fatal disease. 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.
About SD-101
SD-101 is a topical cream that has shown potential to provide improvements in the treatment of severe skin disorders seen in patients of all EB subtypes. Phase II and Phase III studies were conducted in children and adults with EB simplex, recessive dystrophic (RDEB) or junctional EB. A total of 217 EB patients were included in these studies. SD-101 was applied topically to the whole body daily in adults and children from 21 days of age for a period of three months. The primary outcome measure was assessment of target wound closure, in addition to secondary measures, including reduction in blisters and wound body surface area (BSA) coverage.
About Paradigm Therapeutics
Paradigm Therapeutics is a private clinical stage pharmaceutical company focused on developing innovative therapies to address critical medical needs in the treatment of rare diseases. Paradigm's primary focus is the treatment of epidermolysis bullosa (EB). SD-101, their leading therapy for the treatment of skin effects in patients with epidermolysis bullosa, has completed Phase II and Phase III clinical trials. SD-101 is a topical cream that has shown potential to provide improvements in the treatment of severe skin effects seen in patients of all EB subtypes.
Company Contact Dr. Robert Ryan CEO ryan@paradigmtherapeutics.com 919-274-0703 www.paradigmtherapeutics.com
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