FDA Grants Cabaletta Bio Fast Track Designation for Treatment of Mucosal Pemphigus Vulgaris

The U.S. Food and Drug Administration has granted Fast Track designation for Cabaletta Bio’ DSG3-CAART cell therapy for the treatment of rare autoimmune condition mucosal pemphigus vulgaris.

Photo: David Chang, chief medical officer of Cabaletta

Pemphigus vulgaris (PV) is a blistering disease that is characterized by the loss of adhesion between cells of the skin or mucous membranes. PV is caused by the production of autoantibodies that disrupt structural proteins within the skin and/or mucosa that connect with other proteins to enable the skin and/or mucosal cells to connect with each other. The autoantibodies can target desmoglein 3 (DSG3) and/or desmoglein 1 (DSG1), which are primarily expressed in the mucosal membranes and skin, respectively. Mucosal pemphigus vulgaris (mPV) is characterized by autoantibodies against DSG3 only.

Chimeric autoantibody Receptor (CAAR) T cells are designed to selectively bind and eliminate only disease-causing B cells, while sparing the normal B cells that are essential for human health. Cabaletta Bio’s DSG3-CAART is designed to target the cause of mPV, B cells that express pathogenic autoantibodies directed against the DSG3 protein, while preserving normal B cell immune function.

The company plans to initiate a phase 1 trial to evaluate the safety and tolerability of DSG3-CAART in relapsed and/or refractory patients in 2020 with the aim of improving the healing of blisters in patients with mPV. DSG3-CAART is based on technology licensed from, and developed in collaboration with, the University of Pennsylvania.

“The Fast Track designation represents an important next step in our clinical development plans,” said David Chang, 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 Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions that have the potential to address unmet medical needs. A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application review.

The FDA has also granted Orphan Drug designation for DSG3-CAART as a treatment for mPV.

Author: Rare Daily Staff