US FDA Grants Orphan Drug Status to TXA127 for Recessive Dystrophic Epidermolysis Bullosa

The U.S. Food and Drug Administration has granted Orphan Drug Status to TXA127 as a potential treatment for the rare genetic skin disorder Recessive Dystrophic Epidermolysis Bullosa (RDEB).

TXA127 developer, Tarix Orphan LLC, is also partnering with DEBRA Austria, a research collaboration aimed at furthering the development of TXA127 as a potential treatment for RDEB.  Under the agreement, DEBRA will fund a preclinical study of TXA127 that is designed to definitively show the efficacy of TXA127 in a mouse model of RDEB.

TXA127, a pharmaceutical formulation of the natural Angiotensin (1-7) peptide, interferes with the TGF beta pathway, which is involved in the pathophysiology of DEB as well as several other serious orphan diseases. Previous preclinical studies with TXA127 have shown its ability to reduce the fusion of digits, a symptom in RDEB mice that is analogous to mitten deformity common in patients with severe RDEB.