Drug intelligence / Profile preview

allantoin

Development stage
Phase 3
Lead developer
Paradigm Therapeutics
Modality
Small Molecules
Administration
Topical
01

Overview

Zorblisa (SD-101) is a proprietary topical cream formulation of allantoin (6%) that was developed for the treatment of Epidermolysis Bullosa (EB), a group of rare genetic connective tissue disorders characterized by extremely fragile skin and recurrent blister formation. The drug was designed to promote wound healing and skin closure by stimulating the migration and proliferation of fibroblasts and epithelial cells, while also providing anti-inflammatory and keratolytic effects. Originally developed by Scioderm, Zorblisa received FDA Breakthrough Therapy and Orphan Drug designations. Following the acquisition of Scioderm by Amicus Therapeutics in 2015, the drug entered Phase 3 clinical trials. However, in 2017, the pivotal Phase 3 ESSENCE study failed to meet its primary endpoints of complete target wound closure, leading to the discontinuation of the Zorblisa development program.

Brand names
Zorblisa
Other names
SD-101SD101SD 101SD-101-6.0SD101-6.0SD 101-6.0
02

Targets

TLR9 (Toll-like receptor 9)

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