RARE Daily

FDA Grants Rare Pediatric Disease Designation to Ascentage Pharma for Neuroblastoma Therapy

March 22, 2022

The U.S. Food and Drug Administration granted Ascentage Pharma Rare Pediatric Disease designation for alrizomadlin, its experimental therapy for neuroblastoma.

Neuroblastoma is a serious condition that can be life-threatening to pediatric patients. Patients diagnosed as low risk usually have a good prognosis. However, those diagnosed with high-risk disease are difficult to cure, with a large proportion of these patients eventually experiencing disease recurrence. Despite intense multimodal treatments, patients with high-risk neuroblastoma have a poor prognosis with an event-free survival of less than 50 percent. Patients with relapsed or refractory neuroblastoma are extremely difficult to cure, and there is no standard treatment for these patients. According to the American Cancer Society (ACS), there are about 700 to 800 new cases of neuroblastoma each year in the United States. Due to its aggressive nature and high risk of metastasis, neuroblastoma accounts for up to 15 percent of all deaths caused by pediatric cancers.

Alrizomadlin is an orally administered, selective, small-molecule inhibitor of the MDM2 protein. Alrizomadlin has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 protein-protein interaction. In earlier studies, APG-115 as a single agent has shown antitumor activity in in vitro and in vivo models of neuroblastoma, demonstrating a mechanism of action that supports the clinical development of the drug candidate in patients with neuroblastoma.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States. If a New Drug Application in the United States for alrizomadlin is approved, Ascentage may be eligible to receive a Priority Review Voucher from the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, BioMarin Pharmaceutical sold its voucher for $110 million.

Ascentage said it will initiate the clinical study in neuroblastoma as soon as possible.

Author: Rare Daily Staff

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