China News Service, Boao, August 16th. Recently, Qian Xiaowen, deputy director of the Department of Hematology, Pediatrics Hospital of Fudan University, prescribed the first prescription for DANYELZA® (Naxitumumab) in Mainland China. A child about 2 years old was in Hainan. The Lecheng Branch of Provincial Women's and Children's Medical Center became the first patient in Mainland China to use humanized anti-GD2 monoclonal antibody to treat neuroblastoma.

As a highly anticipated immunotherapy innovative drug in the field of neuroblastoma treatment, nalcistuomab brings a new choice for patients with relapsed/refractory high-risk neuroblastoma in my country.

　　Neuroblastoma is one of the most common childhood tumors, accounting for 8% to 10% of all childhood tumors, and is known as the "king of childhood tumors".

The disease site is common in the nerve tissues of the adrenal glands, neck, chest, abdomen and pelvis.

Due to the concealment and metastasis of the primary site, not only the early diagnosis is difficult, but about half of the children are in a high-risk state when they are diagnosed.

Even after comprehensive treatment, the prognosis of children with high-risk neuroblastoma is still poor, with a long-term survival rate of less than 50%.

High-risk children urgently need more effective treatments to improve long-term survival.

　　A few days ago, led by the Children's Hospital of Fudan University, and in cooperation with the Hainan Women and Children's Medical Center, successfully completed the first domestic drug administration of nacistomab immunotherapy in Lecheng, Boao.

On the morning of August 7, the child Yueyue (a pseudonym) received the first intravenous infusion of nacibutuzumab. It is understood that she was able to play with toys and watch videos on her parents’ mobile phones that afternoon.

On the morning of August 9th and 11th, as Yueyue completed the second and third infusions, the first treatment cycle ended smoothly.

　　Yueyue’s father, Mr. Tong (pseudonym), introduced that at the end of 2019, when the child was 5 months old, he found symptoms such as lumps in the abdomen and asymmetry in the size of the eyes. He was diagnosed with neuroblastoma stage 4 high-risk type, with liver metastases and orbits. Bone metastasis is very difficult to treat.

"The child has received chemotherapy, surgery, and radiotherapy and other conventional treatments. It is quite guilty. Although the current situation is relatively stable, the doctor also communicated with us, and the risk of recurrence is very high." Mr. Tong said.

　　From the patient exchange group, Mr. Tong learned about the new trend of foreign neuroblastoma immunotherapy, and was fortunate to have successfully applied for the acceptance of the domestically unlisted immunotherapy innovative drug Naxito at Lecheng Branch of Hainan Women’s and Children’s Medical Center. The first injection of monoclonal antibody rekindled the confidence of a family in treatment.

Mr. Tong said that he also hopes that through Boao's clinical experience, innovative immunotherapy drugs for childhood neuroblastoma will be introduced into the country and included in medical insurance as soon as possible, so that more families with children will benefit.

　　In this regard, Zhang Weiling, chief physician of the Pediatrics Department of Beijing Tongren Hospital, Capital Medical University, said that for middle- and high-risk children, the prognosis is still relatively poor after comprehensive treatment, as if an "untimed bomb" has been implanted in the body. The risk of recurrence.

For parents, special attention should be paid to early symptoms such as decreased appetite, weakness, fever, and joint swelling and pain in their children, early diagnosis and active diagnosis and treatment.

　　Zhai Xiaowen, deputy dean of the Pediatric Hospital of Fudan University, leader of the Department of Hematology, and dean of the Hainan Branch of the Pediatric Hospital of Fudan University (Hainan Children’s Hospital), said that in addition to the treatment methods such as surgery, chemotherapy, radiotherapy, and stem cell transplantation, domestic use There are limited methods and drugs to treat high-risk neuroblastoma, and children still have a high risk of recurrence, and the long-term treatment effect and survival are not satisfactory.

With the international application of immunotherapy drugs, the long-term survival rate of these children has been substantially improved.

　　Dong Kuoran, deputy secretary of the Party Committee of the Children’s Hospital of Fudan University and director of the Department of Oncology, said that the test data of an international multi-center clinical study (including 22 patients) of Naxituzumab in which the Chinese population accounts for 50% show that: Among patients with relapsed or refractory high-risk neuroblastoma treated with tuuzumab combined with granulocyte-macrophage colony stimulating factor (GM-CSF), 68% (15/22) of the patients showed complete or partial remission after treatment (Objective Response Rate, ORR)-Among them, 59% (13/22) of the patients achieved complete remission; 9% (2/22) of the patients had their tumors shrunk by more than 50%.

　　Fan Lichun, Dean of Hainan Women’s and Children’s Medical Center, said that since the Hainan Women’s and Children’s Center established the International Cooperation Department and Lecheng Branch in the Boao Lecheng International Medical Tourism Pioneer Zone in September 2020, it has relied on Boao Lecheng’s pilot policies. , Has carried out innovative work such as the use of licensed medical devices and real-world data research in the fields of women and children's medicine and rare diseases.

"We attach great importance to the medication of the first child with nectomab, and dispatched the strongest multidisciplinary medical care team in the hospital to successfully implement the diagnosis and treatment based on the remote consultation with the expert team of the Pediatric Hospital Affiliated to Fudan University. Our wish It is to allow all children to benefit from the latest international diagnosis and treatment technology for the first time, restore health as soon as possible, and live happily as other children." Fan Lichun said.

　　Naxitumumab is the world's first approved humanized ganglioside GD2 monoclonal antibody, which works by targeting GD2, which is highly expressed in neuroblastoma.

Its innovation is that after binding to GD2 on the surface of tumor cells, nalcistuomab can cause antibody-mediated cytotoxicity and activate the complement system, thereby killing tumors.

　　Public information shows that before being accelerated by the FDA in November 2020, the drug had obtained priority approval qualification, breakthrough therapy designation and orphan drug qualification granted by the FDA.

In China, Saisheng Pharmaceutical has reached an exclusive license agreement with its original research drug company Y-mAbs Therapeutics, and obtained the exclusive cooperative development and commercialization rights of naxituzumab in the Greater China region, and is the first to land this product in Boao Lecheng Innovative immunotherapy drugs benefit domestic children with neuroblastoma.

　　It is worth mentioning that only more than seven months after the approval of the market in the United States, in July 2021, Saisheng Pharmaceutical has formally submitted an application for the marketing authorization of Naxituzumab to the National Medical Products Administration of China and was accepted.

It is reported that Saisheng Pharmaceutical has made a reasonable layout in terms of production and supply, in order to bring hope of treatment to Chinese children more efficiently.

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