CAMBRIDGE, Mass. & BEIJING & HEMEL HEMPSTEAD, England– BeiGene, Ltd. (NASDAQ:BGNE; HKEX:06160) and EUSA Pharma (UK), Ltd. today announced that the China National Medical Products Administration (NMPA) has granted QARZIBA® (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory (R/R) neuroblastoma with or without residual disease. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA).i
“Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. “For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.”
“We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients,” said Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma.
The approval of dinutuximab beta in China for the treatment of patients with high-risk neuroblastoma was supported by clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting. In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls (p<0.01) and five-year overall survival (OS) rate was 64% vs. 50% (p≤0.0001).ii The safety of dinutuximab beta has been evaluated in 514 patients. The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.
