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PHILADELPHIA – Cabaletta Bio, Inc. (NASDAQ:), a clinical-stage biotech company, announced today that its investigational therapy, CABA-201, received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for systemic sclerosis (SSc) treatment.
This autoimmune disease, affecting fewer than 200,000 individuals in the U.S., causes progressive skin and organ fibrosis, leading to a median survival of 12 years post-diagnosis.
The company’s lead product candidate, CABA-201, is a 4-1BB-containing fully human CD19-CAR T cell therapy, currently undergoing clinical trials. The RESET clinical trial program, which includes four Phase 1/2 trials, is evaluating CABA-201 for multiple autoimmune diseases driven by B cells.
The RESET-SSc trial is an open-label study designed to assess the therapy’s efficacy in patients with severe systemic sclerosis across two cohorts. Participants will receive a one-time infusion of CABA-201 following a standard preconditioning regimen.
CABA-201 aims to deplete CD19-positive B cells, potentially resetting the immune system and offering a durable remission without continuous therapy. The drug has also received FDA clearance for Investigational New Drug (IND) applications for other autoimmune conditions, including systemic lupus erythematosus, myositis, and generalized myasthenia gravis.
Orphan Drug Designation provides benefits such as clinical trial tax credits, waived FDA fees, and potentially seven years of marketing exclusivity. This designation is part of the FDA’s commitment to supporting the development of treatments for rare diseases.
Cabaletta Bio specializes in engineered T cell therapies for autoimmune diseases. The company’s CARTA strategy, which includes CABA-201, forms part of its broader CABA platform to develop potentially curative treatments for a range of autoimmune diseases. The information provided is based on a press release statement from Cabaletta Bio.
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