Novartis Expands CIIE 'Circle of Friends,' Partners with PNH Patient Community to Empower Patients and Shape a Healthier Future

ShanghaiNovember 7, 2023PR Newswire -- At the 6th China International Import Expo, Novartis China announced a deep collaboration with the PNH Patient Home in the field of PNH (Paroxysmal Nocturnal Hemoglobinuria), establishing a model for cooperation between multinational pharmaceutical companies and domestic patient organizations in the area of patient communication. As another significant practice in fulfilling its commitment to being "patient-centered," Novartis will systematically communicate and interact with patients through the PNH Patient Home. It is dedicated to identifying pain points in the patient journey, better addressing actual needs, promoting disease awareness, enhancing access to new drugs, reducing the disease burden, empowering patients in their long-term battle against this rare disease with confidence and care, and envisioning a healthier future.

From left to right: Yunlan Xu, Vice President of Novartis China and Head of Communications and Patient Engagement; Qiong Yan, Vice President of Novartis China and Head of Oncology; Jiajia, Founder of PNH Care; Wei Wang, Head of Hematology at Novartis China Oncology.

Meeting Patients' Real Needs, Working Together to Redefine the Patient Healthcare Experience

Based on this collaboration, Novartis will invite patient representatives from the PNH Patient Club active across China to act as consultants and establish a PNH Patient Advisory Committee. This committee will regularly discuss and collect patient feedback and opinions to provide references for future product and service innovations. This is also the first time that Novartis has established a patient advisory committee in a single disease area in China. At the same time, Novartis will fully support the routine work of the PNH Patient Club in areas such as science popularization education and patient care, helping medical professionals, patients, and the entire society build correct awareness of the disease and assisting in improving the diagnosis and treatment management level of the condition. At the end of this month, Novartis will support the PNH Patient Club in carrying out its first-ever Patient Care Day event. In the future, both parties will continue to jointly explore more diversified and sustainable cooperation models to bring truly valuable changes and care to patients.

Jiajia, the founder of the PNH Patient Support Group, stated: "The PNH Patient Support Group is an organization that has grown and developed from within the patient community. We deeply understand the pain points and needs of patients and are committed to transforming PNH from being 'difficult to diagnose and treat' to 'diagnosable and treatable.' The achievements of Novartis in innovative drug development and patient care are evident to all. Today, we are very pleased to announce our collaboration with Novartis, marking a new chapter in serving the PNH patient community in China. In the future, we will work hand in hand with Novartis to provide broader and more targeted services and support to more patients, improving their quality of life and moving together towards a healthier future."

Zhang Ying, President of Novartis China, stated: "I am very pleased to establish cooperation with the PNH Patient Home, listen to the voices of patients, and in turn, benefit the patients. Focusing on patients, shortening and improving the patient journey, and reshaping the patient healthcare experience have always been one of Novartis' strategic priorities and our consistent original intention. This collaboration with the PNH Home is another innovative attempt by Novartis to empower patients together with the pharmaceutical ecosystem. We look forward to continuing systematic and ongoing communication with patients throughout the entire product lifecycle, promoting innovative R&D and drug accessibility, providing multi-faceted support to patients, and contributing Novartis' strength to building a Healthy China."

PNHA Glimmer of Hope in Treatment Illuminates Patients' Lives

PNH is a rare blood system disease, commonly seen in people aged 30-40.^[1]The main clinical manifestations are intravascular hemolysis, potential hematopoietic failure, and thrombotic tendency.^[2], which was included in China's "First List of Rare Diseases" in 2018. In actual clinical diagnosis, due to the low incidence of PNH, it is often overlooked, frequently leading to missed diagnoses and misdiagnoses.^[3]In clinical practice, PNH has also been lacking effective therapeutic drugs. The marketed C5 complement inhibitors, due to persistent C3-mediated extravascular hemolysis^[4]Some patients still cannot摆脱输血依赖, and at the same time, intravenous and subcutaneous injections also affect the quality of life of patients.

Professor Han Bing from the Department of Hematology at Peking Union Medical College Hospital stated: "Despite the remarkable progress in PNH treatment in China in recent years, patients still often face challenges such as difficult diagnosis, limited support, and high medication costs. Once diagnosed, it not only affects the patient's physical health but also casts a significant shadow over their mental well-being and family life. This collaboration between Novartis and the PNH Patient Support Group builds a bridge for multi-party communication and holds substantial importance for innovative developments in the PNH disease field. We look forward to the joint efforts of pharmaceutical companies, patient organizations, and medical institutions enabling patients to gain the possibility of extended life and a better quality of living."

June 2023, The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has officially accepted the marketing authorization application for iptacopan (LNP023) capsules, a novel drug developed by Novartis, and included it in the priority review process. Its proposed indication is for the treatment of adult patients with PNH. Iptacopan, an oral inhibitor targeting the complement alternative pathway B factor developed by Novartis, can not only inhibit intravascular hemolysis and extravascular hemolysis, but its oral administration also improves patient compliance and enhances the medication experience. This breakthrough drug is expected to disrupt the current treatment landscape, offering PNH patients a new therapeutic option and helping them regain health and hope.

Statement: 1. Iptacopan has not yet been approved in mainland China. 2. The purpose of this material is to provide relevant knowledge in the field of diseases and improve disease awareness, not for advertising purposes. 3. The information involved in this material is for reference only; please follow the advice or guidance of doctors or other healthcare professionals.

[1] Rare Disease Advisor. Paroxysmal Nocturnal Hemoglobinuria (PNH)

[2] Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition).

[3] Wang Hongli. Diagnosis of Paroxysmal Nocturnal Hemoglobinuria[J]. Theory and Practice of Diagnostics, 2014, 13(01): 4-6.

[4] EHA Library. Risitano A. 06/08/2023; 387178; PB2063.