Koselugo® (Selumetinib Sulfate Capsules) Successfully Launched in China for Pediatric Patients with Neurofibromatosis Type 1 and Plexiform Neurofibromas

The conference opened with deeply moving personal stories from numerous children with NF1 and their parents. Immediately following was Beijing Union Medical College Hospital.Professor Zhang ShuyangIn the speech, gratitude was expressed to AstraZeneca for its long-term dedication to the rare disease field and commitment to addressing the clinical needs of rare disease patients. Professor Zhang Shuyang stated that in recent years, China's rare disease sector has seen leapfrog development, which is inseparable from the care and support from the state and the CPC Central Committee for rare disease patients, as well as the relentless efforts of medical professionals and all sectors of society. Koselugo^®The approval and launch in China have brought new hope to Chinese pediatric patients, and we look forward to future collaboration with Kesaiyou.^®To further explore the issues in the rare disease field, seek innovative solutions, and provide patients with more comprehensive and higher-quality medical services.

Speech Expert: Professor Zhang Shuyang

Executive President of the China Rare Disease AllianceMr. Línkāng LǐIt was noted that in recent years, there has been significant progress in the diagnosis and treatment of rare diseases in China. Koselugo® has brought hope and promise to patients with rare diseases in China. Moving forward, we need to strengthen clinical training to identify patients earlier, enabling them to access Koselugo sooner, faster, and more effectively.^®。

Speech Expert: Mr. Likang Li

Shanghai Ninth People's Hospital, School of Medicine, Shanghai Jiao Tong UniversityProfessor Li QingfengAs Koselugo^®The PI of the clinical trial stated that the successful launch of Cosentyx® is a significant event in the history of rare disease treatment in China, and expressed honor in witnessing this historic moment with everyone. He pointed out that Cosentyx^®The emergence has lit up a beacon on the life path of children with NF1. Doctors have no regrets, and their medical dedication is wholehearted. It is hoped that with everyone's joint efforts, this drug can be more widely applied.

Speech Expert: Professor Li Qingfeng

Beijing Tiantan Hospital, Capital Medical UniversityProfessor Liu PinanIt was remarked that the situation of children with NF1 and their families is deeply moving, and their pursuit of health is inspiring. We believe that with the support and efforts from all parties, we can build a bright future together with children suffering from NF1.

Speech Expert: Professor Liu Pinan

Senior Vice President of AstraZenecaMs. Soraya BekkaliFirst expressed a warm welcome to the attending experts. Then pointed out that AstraZeneca has a large team of professionals in rare diseases and is always committed to providing innovative solutions for patients with rare diseases, improving drug accessibility to change patient outcomes. Currently, Koselugo® has been approved for marketing in many countries around the world, bringing hope to children with NF1. Ms. Soraya Bekkali said she was very pleased to join everyone in congratulating its launch in China. Meanwhile, the AstraZeneca Rare Disease Team feels a great sense of responsibility and urgency, and will accelerate clinical research and pipeline development as soon as possible, aiming to provide better drugs for more patients.

Speech Expert: Ms. Soraya Bekkali

Speech Expert: Mr. Wang Lei

Vice President of AstraZeneca China, Head of Rare Disease DivisionMs. Hu YiqingIt was stated that in the past year, Koselugo® has achieved a "triple jump" of obtaining approval, commercial launch, and inclusion in medical insurance. In the future, our team will focus on establishing NF1 provincial regional diagnosis and treatment centers, helping doctors in prefecture-level and county-level cities to enhance NF1 disease education, facilitating the early detection of NF1 patients so they can receive the best treatment; assisting experts in building patient databases for further research and exploration, enabling China's innovation to reach the international market. Finally, we wish this conference a complete success.

Speech Expert: Ms. Hu Yiqing

During the exciting product launch ceremony,Professor Zhang Shuyang, Professor Li Lin Kang, Professor Li Qingfeng, Professor Liu Pinan, Ms. Soraya Bekkali, Ms. Hu Yiqing, Mr. Huang Bin, Vice President of AstraZeneca ChinaJointly Participate in the KOSeluzumab Launch^®The listing celebration ceremony, together with experts and peers both on-site and online, jointly witnessed the launch of Cosyoo.^®A Historic Moment in China: Embarking on a New Journey for NF1 Diagnosis and Treatment.

Product Launch Ceremony

Host Expert: Professor Qingfeng Li

First, AstraZeneca Global Senior Vice President, Head of Rare Disease Drug Clinical DevelopmentProfessor Christophe HotermansComprehensive sharing of AstraZeneca's rare disease R&D pipeline and Cosentyx^®Latest Advances in Clinical Research. In the past few years, more than 10,000 rare diseases have been discovered, and 90% of patients currently have no available treatment. Cosentyx^®The successful launch has benefited more than 2,500 children with NF1 worldwide, and more drug-related studies are currently underway. In order to provide earlier, more precise, and more effective treatment for more patients, AstraZeneca will continue to work closely with academia and drug regulatory agencies, overcoming challenges to improve the future treatment landscape for rare diseases.

Sharing Expert: Professor Christophe Hotermans

Subsequently, Xuanwu Hospital of Capital Medical UniversityProfessor Hao Wu"NF1 as I See It: The Current State of the Disease" is vividly illustrated through multiple NF1 patient cases. As a complex condition with high surgical difficulty and "zero" sense of accomplishment, NF1 leads to reduced life expectancy, increased hospitalization rates, heavier economic burdens, and a significant decline in patients' quality of life across various domains. Between 30% to 50% of NF1 patients develop plexiform neurofibromas (PN), which tend to grow continuously over time and are often accompanied by severe clinical complications. Koselugo^®The availability of the drug means that such patients no longer have no treatment options, which can be said to be a major breakthrough in the field. In addition, the treatment of NF1-PN still requires more multi-party cooperation, disease awareness, and standardized treatment protocols. Every small advancement is crucial for millions of families. It is hoped that more people will come to understand NF1, and that more children with the condition will achieve better therapeutic outcomes.

Sharing Expert: Professor Wu Hao

Subsequently, the Asan Medical Center in South KoreaProfessor Beom Hee LEEFor Cosentyx^®Prof. Beom Hee LEE delivered a brilliant sharing of application experience in Korea. He emphasized that the diagnosis of NF1 requires the joint efforts of a strong multidisciplinary (MDT) team and stated that for NF1-PN patients aged 3 to 18, Koselugo^®The treatment effect is excellent, which can significantly reduce tumor volume, improve patients' quality of life and cognition, relieve their pain, and the adverse reactions are manageable and controllable. Currently, Kosaiyou^®In South Korea, it is also covered by health insurance, allowing children over the age of 3 to benefit from it.

Sharing Expert: Professor Beom Hee LEE

Immediately following, Peking Union Medical College HospitalProfessor Yicheng ZhuIn-depth Interpretation of the "Multidisciplinary Diagnosis and Treatment Guidelines for Type Ⅰ Neurofibromatosis (2023 Edition)". NF1 patients present with varying ages of onset, diverse clinical manifestations, and involvement of multiple systems, necessitating multidisciplinary collaboration to assess and develop MDT diagnosis and treatment plans. Long-term regular follow-up and periodic health monitoring are also required. Professor Zhu Yicheng stated that drug therapy provides a new option for NF1 patients, and the establishment of a well-functioning MDT team can enhance clinical diagnostic and treatment standards. With advancements in disease diagnosis and treatment as well as innovative drug research, overcoming NF1 is within reach.

Sharing Expert: Professor Yicheng Zhu

Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of MedicineProfessor Wang ZhichaoThen shared Cosentyx^®The latest data from clinical research and real-world cases in China. The SPRINT Phase Ⅰ study determined 25mg/m² bid as the optimal dose for Coseluz.^®The maximum tolerated dose, tumor volume in PN pediatric patients reduced by 31%, with an objective response rate (ORR) reaching 71%; the ORR in the SPRINT Phase II study was as high as 68%, 82% of pediatric patients maintained remission for over 12 months, and the 3-year progression-free survival (PFS) rate was up to 84%. One 10-year-old boy participating in the study saw his tumor shrink by 36.2% after one year of treatment, and by the time he turned 16, he had achieved a very good quality of life. Long-term follow-up data from the two studies showed that the drug's efficacy is durable with a good safety profile. Chinese data indicates that Koselugo® is rapidly absorbed by pediatric patients, with an ORR reaching 63%, and all patients showed targeted PN reduction, with 88% of pediatric patients experiencing a tumor size reduction ≥20%. Adverse reactions were mostly Grade 1-2. Currently, multiple multidisciplinary treatment projects focused on NF1 are ongoing, with the hope that China’s experience will not only serve Chinese patients well but also benefit patients globally.

Sharing Expert: Professor Wang Zhichao

The subsequent roundtable discussion session was held inProfessor Li Qingfengunder the leadership of.Professor Liu Pinan, Guangzhou Women and Children's Medical CenterProfessor Liu Li,Professor Yicheng Zhu、Professor Wang ZhichaoJointly on how to standardize Cosentyx^®The clinical application and the acceleration of evidence-based data accumulation were discussed in depth. Experts expressed that clinical practice needs to overcome resistance, establish more excellent MDT teams, and provide more scientific guidance and humanistic care to patients and their families; the establishment of biobanks requires substantial manpower and resources, and clinical practice needs to make better use of hospital resources; drug accessibility and disease awareness are also important aspects that need special attention and efforts in clinical practice.

Roundtable Discussion Session

Professor Liu PinanIn the speech, it was noted that NF1 is an extremely complex disease with as many as twenty to thirty clinical symptoms, involving multiple disciplines such as radiology, neurosurgery, and dermatology. We should not be afraid of difficulties, continuously deepen the clinical understanding of NF1, strengthen multidisciplinary cooperation, gradually implement diagnostic and treatment standards, and improve the level of clinical diagnosis and treatment.

Host Expert: Professor Liu Pinan

Immediately following, the Capital Medical University Affiliated Beijing Children's HospitalProfessor Xiaoling WangFirst, from a pharmaceutical perspective, the exploration journey of Koselugo® in children with NF1 was shared. Based on positive clinical data, Koselugo^®Recommended as the standard treatment drug for pediatric NF1-PN indications by multiple guidelines both in China and internationally, providing available treatment options for children. The treatment of rare diseases is a systematic project that requires joint efforts from all parties. We hope that through our collective efforts, we can promote the implementation of policies and contribute to "Healthy China."

Sharing Expert: Professor Xiaoling Wang

Thereafter, Shandong Provincial HospitalProfessor Wang GuodongShared the development journey of the NFI-PN diagnosis and treatment model in Shandong. The multidisciplinary team (MDT) at Shandong Provincial Hospital was established in March 2023. After continuous optimization, a specialized NF1 MDT clinic has been set up, along with activities such as online platform exchanges, free clinics, and disease awareness campaigns, providing efficient and personalized services to patients. The establishment of the Shandong Rare Disease Medical Quality Control Center and the NF1 Special Disease Group further promotes the upgrading of diagnosis and treatment as well as patient reporting, enabling early diagnosis and treatment for patients and helping more patients gain a new lease on life.

Sharing Expert: Professor Guodong Wang

Peking Union Medical College HospitalProfessor Feng FengThe imaging evaluation of NF1 was discussed in detail. Magnetic resonance imaging (MRI) is the preferred imaging method for NF1, and baseline whole-body MRI is clinically recommended, especially for NF1 patients in late adolescence or early adulthood (the transitional period from childhood to adulthood). Moreover, whole-body MRI is more advantageous for detecting multiple or larger pNFs that extend beyond traditional anatomical planes. Of course, in some cases, computed tomography (CT), positron emission tomography (PET), and ultrasound may also be used as auxiliary examinations. Additionally, it is expected that more histopathological and tumor burden-related tests will be incorporated to guide clinical treatment.

Sharing Expert: Professor Feng Feng

Subsequently, Xiangya Hospital of Central South UniversityProfessor Peng JingShared pediatric experience in the diagnosis and treatment of NF1. Professor Peng Jing stated that knowledge dissemination comes first, prevention is key, and society should increase its attention to rare diseases while investing more in humanistic care. NF1 has a complex disease profile, and children often face misdiagnosis or multiple referrals at their initial visit. NF1 carries a high risk of malignancy, with malignant changes potentially occurring at any point during the patient's lifetime. Early identification is crucial, followed by selecting surgical treatment or MEK inhibitor targeted therapy based on individual health conditions and personal preferences. Current data suggests that Koselugo...^®The efficacy is remarkable, and the adverse reactions are manageable and controllable, providing a powerful treatment option for clinical practice. The first case was enrolled in Cosentyx.^®Patients in Phase Ⅳ study showed significant improvement in symptoms after more than twenty days of medication, greatly enhancing the treatment confidence of both the children and their families. Of course, NF1 requires long-term management, and we will continue to monitor the changes in the children's conditions.

Sharing Expert: Professor Peng Jing

Children's Hospital Affiliated to Shandong UniversityProfessor Wang GuangyuProfessor Wang Guangyu comprehensively introduced the NF1 diagnosis and treatment experience of his hospital from the perspective of pediatric neurosurgery. Professor Wang Guangyu stated that the disease burden of NF1-PN is severe, with a high risk of malignancy, and the targeted drug Koselugo.^®As the world's first and currently only approved therapeutic drug, it has shown good efficacy and filled the gap in clinical drug treatment. Since the establishment of the NF Special Disease Clinic at the hospital in August 2023, 27 patients have been treated, including a 3-year-and-1-month-old boy who came to the hospital for treatment 1 year and 6 months after discovering a mass on the right side of his face. The family reported that after using Koselugo...^®During the treatment, the facial mass shrank at a visibly rapid rate. Currently, the child is under further follow-up.

Sharing Expert: Professor Wang Guangyu

The First Hospital of China Medical UniversityProfessor Xugang XuFrom a dermatological perspective, shared experiences in treating NF1. Professor Xu Xuegang stated that skin involvement in NF1 mainly includes café-au-lait spots, freckles, and neurofibromas, and clinical attention should be paid to differential diagnosis. Treatment options such as surgical excision and laser therapy address symptoms but not the root cause, and treatment should be tailored to the patient’s needs; Koselugo^®Targeted therapy may offer more thorough treatment for a greater number of NF1-PN patients. For instance, an 11-year-old girl I treated experienced results with Koselugo.^®After one year of treatment, the café-au-lait spots significantly faded, the tumor shrank, and the pain was alleviated.

Sharing Expert: Professor Xuegang Xu

In the roundtable discussion session, inProfessor Liu PinanUnder the leadership of, the Department of Pharmacy at Beijing Children's Hospital, Capital Medical UniversityProfessor Xiaoling Wang, Department of Head and Neck Surgery, Beijing Children's Hospital, Capital Medical UniversityProfessor Wang Shengcai, Shandong Provincial Hospital NeurosurgeryProfessor Zhang Zhen, Department of Neurosurgery, Children's Hospital of Zhejiang University School of MedicineProfessor Shen Zhipeng, Department of Dermatology, First Hospital Affiliated to China Medical UniversityProfessor Xuegang XuExperts shared their understanding and cooperation experience of MDT diagnosis and treatment from the perspectives of pharmacy, neurology, neurosurgery, dermatology, etc. All experts agreed that NF1 should be detected early, treated early, and managed over the long term, emphasizing the important role of the MDT team in NF1 diagnosis and treatment, as well as the significance of improving drug accessibility, and strengthening social attention and support.

Roundtable Discussion

This meeting came to an end amidst enthusiastic discussions.Professor Liu PinanIn conclusion, NF1 is a disease involving multiple systems and disciplines, with complex and diverse clinical manifestations. It requires the participation of more experts to guide the direction of clinical diagnosis and treatment. We also advocate for more people to pay attention to and care for NF1, working together to build a better future for NF1.

Thus, Cosentyx^®The launch event in China was a complete success! Once again, we would like to thank the participating experts for their wonderful presentations and discussions, as well as the attention from peers across China. We look forward to further success.^®Enable more "bubble babies" to benefit and boost the development of NF1 diagnosis and treatment in China.