FDA grants orphan drug designation to cell therapy for Buerger’s disease
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The FDA granted orphan drug designation to CLBS12, an investigational nongene-edited cell therapy for the treatment of Buerger’s disease, according to the agent’s manufacturer.
Buerger’s disease, also known as thromboangiitis obliterans, is a condition related to critical limb ischemia and has no approved treatments in the United States.
CLBS12 (Honedra, Caladrius Biosciences) is an autologous cell therapy derived from CD34+ cells. Patients who receive the therapy undergo apheresis that includes drug-induced mobilization of CD34+ cells from bone marrow. The cells are subsequently isolated, concentrated and administered as an IV infusion.
The process takes 3 to 4 days and avoids the need for bone marrow aspiration of CD34+ cells, according to the manufacturer.
“We are very pleased that the FDA has granted orphan drug designation to CLBS12 in Buerger’s disease,” David J. Mazzo, PhD, president and CEO of Caladrius, said in a company-issued press release. “Without a currently approved or effective treatment for this condition in the U.S., a significant unmet need remains for therapies that slow, stop or, ideally, reverse this debilitating disease.”
CLBS12 is being evaluated for critical limb ischemia and Buerger’s disease in an open-label, phase 2 study based in Japan. Approximately 60% of patients in the cohort with Buerger’s disease are free of critical limb ischemia — one of the study’s endpoints — “despite the natural history of continuous disease progression leading to amputation,” Mazzo said.
“With this designation we can now engage FDA in discussions to define the most efficient and rapid development pathway to registration in the U.S.,” he added. “Achieving orphan designation for CLBS12 takes us one step closer to realizing our goal of fulfilling the unmet medical need for [patients with Buerger’s disease] around the world.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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