An FDA advisory panel will make the call July 12 to recommend for or against agency approval of the first gene therapy. A favorable decision could see the regulator sanction the treatment by early this fall. Drug manufacturer Novartis and the University of Pennsylvania doctors and scientists who tested it are holding out hope for CAR T-cell therapy, which uses patients' own genetically modified immune cells to fight blood cancers. In a multinational trial of pediatric patients, it achieved remission in 83% of participants67% of whom remained in remission 1 year later. While CAR T-cell therapy initially would be offered to children and young adults not responding to standard leukemia treatment, research has shown it to be effective in adults as well. Substantial concerns about safety and cost, however, could sway the FDA committee away from approval. Analysts' projections put the price for a one-time infusion at $300,000$600,000, and there also is the risk of serious adverse effectsincluding neurotoxicity and cytokine release syndrome. Novartis is addressing the safety issue by planning a contained launch of the product, rather than simply unleashing it on the entire market. Under its plan, only 3035 medical centers would be authorized to administer CAR T-cell therapy, most having participated in the clinical trial and all having received extensive training.

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First gene therapy on the cusp of FDA approval - Pharmacy Today, American Pharmacists Association, pharmacist.com

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