Paradigm Therapeutics Inc., a biopharmaceutical company, has successfully finalized the acquisition of the global rights for SD-101, a topical whole-body treatment for all subtypes of Epidermolysis Bullosa (EB), from Amicus Therapeutics. The specifics of the agreement have not been publicly disclosed.

SD-101 has earned the Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for the comprehensive treatment of EB. Alongside this, SD-101 has also been granted Orphan Drug designation from both the FDA and the European Commission (EC), and notably, it was the first treatment for EB to receive the FDA's Breakthrough Therapy designation.

Robert Ryan, Ph.D., Chief Executive Officer of Paradigm Therapeutics, expressed enthusiasm regarding the prospects of further developing SD-101 and initiating discussions with the FDA to expedite the path towards registration for this therapy across all EB subtypes. Dr. Ryan acknowledged the substantial need for innovative treatment options to address the distressing skin effects experienced by Epidermolysis Bullosa patients, underscoring Paradigm Therapeutics' commitment to providing a new therapeutic solution for a condition with limited effective whole-body treatments.

Dr. Ryan emphasized, "Epidermolysis Bullosa is a profoundly challenging disease. Our clinical data strongly indicate that SD-101 brings beneficial effects to a broad range of EB patients, compelling us to advance towards registration. Results from Phase II and Phase III trials have shown that SD-101 is a potential candidate for whole body treatment of skin lesions and wounds in both pediatric and adult EB patients lacking alternatives. Moreover, the results highlighted the favorable safety profile of SD-101."

About Epidermolysis Bullosa (EB): Epidermolysis bullosa is a rare genetic disorder characterized by extremely fragile skin that leads to severe blistering and open wounds, often manifesting from birth. No approved therapies currently exist to treat the entire body of EB patients, impacting numerous individuals from childhood through adulthood. The primary standard of care involves pain management, wound cleaning, and bandaging to prevent infections. EB is a chronic condition that can result in disfigurement and even fatality. While there are various genetic and symptomatic forms of EB, all share the common symptom of fragile skin that blisters and tears, often triggered by minor friction or trauma.

About SD-101: SD-101 is a topical cream that displays potential in addressing the severe skin disruptions prevalent in patients across all subtypes of EB. Phase II and Phase III studies encompassed children and adults with Simplex, Recessive Dystrophic (RDEB), or Junctional EB. These studies comprised a total of 217 EB patients. Over a three-month period, SD-101 was applied topically to the entire body daily, even in infants as young as 21 days old. The primary endpoint evaluated target wound closure, along with secondary measurements encompassing the reduction in body surface area (BSA) affected by blisters and wounds.

About Paradigm Therapeutics: Paradigm Therapeutics is a privately held clinical-stage pharmaceutical company dedicated to the development of innovative therapies addressing critical medical needs within the rare disease treatment landscape. The company's primary focus centers on treating Epidermolysis Bullosa (EB). The company's leading therapy, SD-101, designed to treat skin effects in EB patients, has successfully concluded Phase II and Phase III clinical trials. SD-101, a topical cream, has demonstrated potential in improving the severe skin effects evident in patients across all EB subtypes.

Company Contact: Dr. Robert Ryan Chief Executive Officer rryan@paradigmtherapeutics.com 919-274-0703 www.paradigmtherapeutics.com

 

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