Cabaletta Bio Says FDA Grants Orphan Drug Designation To CABA-201 To Treat Systemic Sclerosis

(RTTNews) - Biotechnology company Cabaletta Bio, Inc. (CABA) announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc).

CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. The RESET (REstoring SElf-Tolerance) clinical trial program includes four Phase 1/2 trials advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-SSc trial.

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 seven years of marketing exclusivity.