The Food and Drug Administration (FDA) has granted Fast Track designation to DSG3-CAART (desmoglein 3 chimeric autoantibody receptor T cell therapy; Cabaletta Bio), a potential treatment for mucosal pemphigus vulgaris, a rare and life-threatening autoimmune disorder that causes progressive painful blistering.
DSG3-CAART 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. The phase 1 DesCAARTes trial will evaluate the safety and tolerability of the treatment in patients with relapsed and/or refractory mucosal pemphigus vulgaris.
“We believe that this Fast Track designation, coming shortly after the Orphan Drug designation for DSG3-CAART, further demonstrates that [ mucosal pemphigus vulgaris] is a devastating, rare disease for which patients have limited treatment options resulting in a large unmet need,” said David J. Chang, MD, Chief Medical Officer of Cabaletta. “We appreciate the benefits provided by this designation, including the opportunity for increased access to the FDA and potential acceleration of our clinical development path and regulatory review process.”
The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.
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