FDA grants breakthrough therapy designation to burtomab for pediatric neuroblastoma

The FDA granted breakthrough therapy designation to burtomab for the treatment of pediatric patients with relapsed or refractory neuroblastoma who have central nervous system or leptomeningeal metastasis.

“There are no approved drugs to treat this type of advanced neuroblastoma,” Nai-Kong Cheung, MD, PhD, of Memorial Sloan Kettering Cancer Center, who created the antibody, said in a press release. “[Memorial Sloan Kettering] treats more patients with neuroblastoma than any other institution in the world, and with evidence-based treatment approaches like this, we can offer real hope for better treatments to patients and families confronting this devastating disease.”

The FDA based the breakthrough therapy designation on data from a pivotal clinical study of burtomab — licensed to Y-mAbs Therapeutics — in combination with radiolabeled iodine for the treatment of pediatric patients with neuroblastoma that metastasized to the central nervous system or the leptomeninges.

“We are very pleased that the FDA has granted the breakthrough therapy designation to burtomab and thereby shortened the timelines for making this therapy available to the children facing an unmet medical need,” Thomas Gad, founder, president and head of business development and strategy at Y-mAbs Therapeutics, said in the release. “Burtomab clearly provides a potential curative treatment option for pediatric patients otherwise faced with little or no options. This is an important milestone achievement for Y-mAbs, and we continue to work with our strategic partner Memorial Sloan Kettering and the regulatory authorities to advance burtomab to patients suffering from refractory leptomeningeal metastasis from neuroblastoma as quickly as possible.”