Orphan Drug Designation Granted to Treatment of Pemphigus Vulgaris


DSG3-CAART is designed to target the cause of mucosal pemphigus vulgaris while sparing normal B cell populations that provide immunity from infection.

The FDA has granted Orphan Drug Designation to DSG3-CAART (Cabaletta) for the treatment of pemphigus vulgaris (PV). The therapy is designed to target the cause of mucosal PV (mPV).

“For affected patients, despite current treatment options, there is an urgent unmet need for more effective and durable therapies that can provide reliable, complete, and persistent remission from the disease beyond general immune suppression and B cell depletion provided by current treatment options,” said David Chang, MD, chief medical officer of Cabaletta, in a prepared statement.

mPV is a rare and potentially fatal, chronic autoimmune disease characterized by the loss of adhesion between cells of mucous membranes, resulting in widespread damage, painful blisters of the mucosal membranes, and increased susceptibility to life-threatening systemic infections.

Because mPV is categorized by B cells that express pathogenic autoantibodies directed against the desmoglein 3 (DSG3) protein, the DSG3-chimeric auto-antibody receptor T cell (CAART) therapy can selectively target this disease while sparing all other B cell populations that provide beneficial immunity from infection.

“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 to generate and then report acute safety data from the first cohort of patients by the end of 2020,” said Chang in a prepared statement.

The FDA grants Orphan Drug Designation to drugs or biologics intended to treat or prevent rare diseases or conditions that affect fewer than 200,000 individuals in the United States. This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees, and potential eligibility for 7 years of marketing exclusivity.


  • Medication Dose Increased Likeliness of Remission for Patients with Pemphigus
  • Study: Rituximab Superior to Mycophenolate Mofetil as Pemphigus Vulgaris Treatment


FDA Grants DSG3-CAART Orphan Drug Designation for the Treatment of Pemphigus Vulgaris [news release]. Philadelphia, PA; January 29, 2020: Cabaletta Bio. https://investors.cabalettabio.com/news-releases/news-release-details/fda-grants-dsg3-caart-orphan-drug-designation-treatment Accessed January 30, 2020.

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