FDA grants orphan drug designation to CAR T-cell therapy for systemic sclerosis
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Key takeaways:
- The designation provides incentives such as tax credits for trial costs and waived user fees.
- A program including four phase 1/2 studies will examine CABA-201 across multiple autoimmune diseases, including SSc.
The FDA has granted orphan drug designation to Cabaletta Bio’s CAR T-cell investigational therapy for the treatment of systemic sclerosis, according to a press release from the manufacturer.
The drug, currently known as CABA-201, is a 4-1BB-containing, fully human CD19-CAR T cell, Cabaletta said in the release. According to the company, a clinical trial program including four phase 1/2 studies is advancing to examine CABA-201 across multiple autoimmune diseases, including SSc.
“Patients diagnosed with systemic sclerosis, a rare and life-threatening chronic autoimmune disease characterized by progressive skin and internal organ fibrosis, face limited treatment options as current therapies provide only modest effects and focus on treating the complications associated with the disease,” David J. Chang, MD, chief medical officer of Cabaletta, said in the release. “With an average patient survival of 12 years following diagnosis, there is a significant unmet need for new treatment options that focus on eliminating the root cause of the disease to prevent further organ damage for patients.
“Based on the role of B cells and the recently published academic clinical data with CD19-CAR T therapy in systemic sclerosis, we believe CABA-201 may transform the treatment for systemic sclerosis,” he added. “Orphan drug designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we develop CABA-201 for patients with systemic sclerosis.”
The clinical trial program will be known as REstoring SElf-Tolerance, or RESET. According to Cabaletta, the RESET-SSc trial will be a phase 1/2, open-label study of CABA-201 in patients with SSc across two parallel cohorts.
One cohort will include six patients with severe skin involvement, while the other will feature six patients who meet pulmonary, cardiac or renal involvement criteria regardless of skin involvement. Patients will receive a standard preconditioning regimen of fludarabine and cyclophosphamide, followed by a one-time infusion of CABA-201 at a dose of 1x106 cells/kg. The study will evaluate the ability of CABA-201 to “transiently, but fully, eliminate B cells, potentially enabling durable remissions via a “reset” of the immune system,” the release said.
The FDA’s orphan drug designation is granted to therapies intended to treat or prevent rare diseases that impact fewer than 200,000 individuals in the United States. The designation qualifies the drug and manufacturer for multiple incentives, including partial tax credit for clinical trial costs, waived user fees and eligibility for 7 years of marketing exclusivity.
The FDA has so far cleared four investigational new drug applications for CABA-201 in multiple autoimmune conditions, including systemic lupus erythematosus, myositis, SSc and generalized myasthenia gravis. In all, the RESET program will feature a total of nine cohorts advancing simultaneously.
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