FX.co ★ Cabaletta Bio Says FDA Grants Orphan Drug Designation To CABA-201 To Treat Systemic Sclerosis

Biotech firm, Cabaletta Bio Inc., has announced that its investigational therapy, CABA-201, a 4-1BB-containing fully human CD19-CAR T cell, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This decision recognizes CABA-201 as a potential treatment for systemic sclerosis (SSc), a condition driven by B cells which affect fewer than 200,000 individuals in the United States.

The drug is currently under development by the company, part of a wider program known as RESET (REstoring SElf-Tolerance), which incorporates four Phase 1/2 trials evaluating CABA-201 across a range of autoimmune conditions. Included within these is the Phase 1/2 RESET-SSc trial.

The FDA's Orphan Drug Designation acknowledges the drug's potential to prevent or treat rare diseases. This comes with certain benefits for Cabaletta, potentially entitling the company to a partial tax credit related to clinical trial costs, exemption from user fees, and a seven-year exclusive marketing period following FDA approval.