FDA Awards Orphan Drug Designation to Cabaletta’s Innovative Myositis Therapy

Cabaletta Bio

PHILADELPHIA, PA — The biotech firm Cabaletta Bio, Inc. (Nasdaq: CABA) has secured an Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its investigational therapy, CABA-201. This fully human CD19-CAR T cell therapy containing 4-1BB is being developed to treat idiopathic inflammatory myopathies (IIM), also known as myositis.

Myositis is a severe autoimmune disease believed to be driven by B cells. Current treatment options offer limited efficacy, with many patients responding inadequately to available therapies. Given this situation, there is an urgent need for innovative medicines capable of changing the current treatment paradigm.

“CABA-201 is designed to deeply and transiently deplete CD19-positive B cells, which may enable an immune system reset,” said Dr. David J. Chang, Chief Medical Officer of Cabaletta. This approach could potentially offer durable remission off therapy in patients diagnosed with myositis and other autoimmune diseases where B cells play a significant role.

CABA-201 is currently under evaluation across multiple autoimmune conditions in four RESET™ (REstoring SElf-Tolerance) Phase 1/2 trials, including the Phase 1/2 RESET-Myositis™ trial.

The FDA grants ODD status to drugs or biologics that aim to treat or prevent rare diseases affecting fewer than 200,000 individuals in the United States. This designation comes with several potential benefits, including partial tax credits for clinical trial expenditures, waived user fees, and potential eligibility for seven years of marketing exclusivity.

The recognition of CABA-201 as an orphan drug highlights its potential as a groundbreaking treatment for a rare disease. With this designation, Cabaletta is well-positioned to accelerate the development of CABA-201, which could become the first targeted—and potentially curative—cell therapy for patients with autoimmune diseases.

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