PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to its investigational therapy, CABA-201. This designation marks a critical milestone in the development of new treatments targeting the root causes of autoimmune diseases.
CABA-201, a cutting-edge 4-1BB-containing fully human CD19-CAR T cell therapy, is currently under investigation as a potential breakthrough in the treatment of autoimmune diseases driven by B cells. The therapy is part of Cabaletta’s RESET™ clinical trial program, which encompasses four Phase 1/2 trials aimed at evaluating CABA-201’s efficacy across various autoimmune conditions, including systemic sclerosis in the RESET-SSc™ trial.
Systemic sclerosis is characterized by progressive fibrosis of the skin and internal organs, leading to significant morbidity and mortality. Patients diagnosed with this chronic condition often face bleak prospects, with average survival rates lingering around 12 years post-diagnosis. Current treatment modalities offer limited relief, primarily addressing complications rather than targeting the disease’s underlying causes.
David J. Chang, M.D., Chief Medical Officer of Cabaletta, expressed optimism about the potential impact of CABA-201 on systemic sclerosis treatment. “Given the pivotal role of B cells in systemic sclerosis and encouraging data from recent clinical studies involving CD19-CAR T therapy, we are hopeful that CABA-201 could revolutionize the treatment landscape for this devastating disease,” said Chang.
The FDA’s Orphan Drug Designation is reserved for promising drugs and biologics intended to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 individuals in the United States. This status provides several benefits to the sponsor, including tax incentives for clinical research costs, exemption from certain FDA application fees, and the possibility of seven years of market exclusivity upon approval.
The recognition of CABA-201 as an orphan drug underscores the urgent need for innovative therapies that can address the unmet needs of patients with systemic sclerosis. As Cabaletta Bio advances its clinical trials, the company hopes to unlock new pathways to treatment that could halt or even reverse the progression of this life-threatening disease, offering hope to thousands of patients and their families.
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