Pharma Focus Europe

Paradigm Therapeutics Acquires Advanced Late-stage "Breakthrough Therapy" for Treating All Subtypes of Epidermolysis Bullosa (EB)

Wednesday, August 23, 2023

Paradigm Therapeutics Inc., a prominent biopharmaceutical firm, has successfully concluded the acquisition of the global rights to SD-101 from Amicus Therapeutics. SD-101 represents a topical remedy designed to address all subtypes of Epidermolysis Bullosa (EB), an uncommon yet devastating genetic disorder characterized by skin fragility, blistering, and chronic wounds. Although the specific financial terms of the transaction remain undisclosed, this acquisition marks a significant step in therapeutic advancement.

Notably, SD-101 has garnered several distinguished designations from regulatory bodies. These include the Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA), as well as the Orphan Drug designation from both the FDA and the European Commission (EC). Furthermore, it holds the esteemed Breakthrough Therapy designation from the FDA, underscoring its potential therapeutic breakthrough status.

Robert Ryan, Ph.D., Chief Executive Officer of Paradigm Therapeutics, expressed his enthusiasm regarding the next phases of SD-101's development and the collaborative efforts to expedite its regulatory approval process. Dr. Ryan emphasized the pressing need for effective treatments to alleviate the severe dermatological effects of Epidermolysis Bullosa. He reaffirmed the commitment of Paradigm Therapeutics to provide an innovative therapeutic option for patients facing this challenging condition.

Dr. Ryan further highlighted the gravity of Epidermolysis Bullosa as a disease and the promising outcomes indicated by SD-101's clinical data. Notably, the concluded Phase II and Phase III trials showcased encouraging results of SD-101 in treating skin lesions and wounds among pediatric and adult EB patients, particularly in cases where comprehensive treatment alternatives are lacking. These trials also underscored the favorable safety profile associated with SD-101 administration.

Epidermolysis Bullosa entails a distressing journey for those affected, characterized by fragile skin, blistering, and wounds arising from minor friction or trauma. Despite its impact, effective treatments that encompass the entire body of EB patients remain elusive. Presently, the primary focus of medical care centers around pain management, wound cleansing, and meticulous bandaging to mitigate infection risks.

SD-101, a topical cream, emerges as a potential solution to address the pronounced skin disruptions inherent to all forms of EB. The comprehensive Phase II and Phase III trials encompassed individuals spanning different EB subtypes, including Simplex, Recessive Dystrophic (RDEB), and Junctional EB. A collective cohort of 217 EB patients, across various age groups, participated in these trials. Over a three-month period, SD-101 was administered topically on a daily basis, covering the entire body. The primary benchmark for efficacy was centered on wound closure assessments, coupled with secondary measurements gauging reductions in blister and wound coverage across the body's surface.

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