Novel Cell Therapy Gets Orphan Drug Status for Mucosal Pemphigus Vulgaris

The Food and Drug Administration (FDA) has granted Orphan Drug designation to DSG3-CAART (Cabaletta Bio), a potential treatment for pemphigus vulgaris, a rare and life-threatening autoimmune disorder that causes progressive painful blistering of the skin and mucous membranes.

DSG3-CAART, or desmoglein 3 chimeric autoantibody receptor T cell therapy, is designed to selectively target the cause of mucosal pemphigus vulgaris, B cells that express pathogenic autoantibodies directed against the DSG3 protein, while sparing normal B cell immune function.

“Orphan Drug designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we prepare to initiate the DesCAARTes trial,” said David Chang, MD, Chief Medical Officer of Cabaletta.

Last October, the Company received clearance to initiate a first-in-human trial. Safety data from the first cohort of patients are expected sometime toward the end of 2020.

Orphan Drug designation is granted to investigational treatments intended to treat or prevent rare diseases or conditions that affect fewer than 200,000 individuals in the US.

For more information visit cabelettabio.com.