"Pilot Policy" Breakthrough: Isatuximab Becomes First Hematologic Drug in China to Gain NMPA Filing Acceptance Based on Lecheng Real-World Evidence

ShanghaiDecember 12, 2023PR Newswire -- Today, the marketing authorization application for Isatuximab Injection, a next-generation CD38 monoclonal antibody developed by Sanofi, has been officially accepted by the National Medical Products Administration (NMPA). It is intended for use in combination with pomalidomide and dexamethasone to treat adult patients with multiple myeloma (MM) who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor.

As one of the three pilot drugs approved to conduct clinical real-world data research in the Boao Lecheng International Medical Tourism Pilot Zone in Hainan, the Isatuximab injection has become the first hematological tumor treatment drug to have its marketing authorization application accepted by the National Medical Products Administration using real-world data from Lecheng. The Isatuximab injection is a monoclonal antibody that targets a specific epitope of the CD38 receptor on multiple myeloma cells, triggering various mechanisms of action, including programmed tumor cell death (apoptosis) and modulation of the body's immune response. It is expected to open new avenues for the treatment of patients with relapsed or refractory multiple myeloma.

Jia Ning, Secretary of the Party Committee and Director of the Management Bureau of Lecheng Pilot Zone, stated: "The acceptance of the marketing authorization application for Isatuximab injection represents a significant breakthrough in real-world research in the field of hematological oncology. As the only 'medical special zone' in China, Lecheng Pilot Zone leverages its unique policy of 'pioneering trials' to conduct cutting-edge real-world data application research internationally. This effort accumulates experience and explores methods for the reform of the medical device review and approval system in China, providing new pathways and solutions to accelerate the accessibility of innovative global pharmaceuticals and medical devices in China. In the future, Lecheng will utilize its policy advantages and the aggregation of medical resources to attract more innovative and high-quality pharmaceuticals and medical devices to land in Lecheng, fully promoting the advancement of real-world research work, and allowing more medical technology innovation achievements to benefit Chinese patients more quickly."

In June 2019, the National Medical Products Administration and the Hainan Provincial Government jointly launched the Hainan Clinical Real-World Data Application Pilot Program, making Lecheng Pilot Zone the first area in China to carry out a clinical real-world data application pilot. Since 2020, guidelines such as the "Guiding Principles on the Use of Real-World Evidence to Support Drug Research and Evaluation (Trial)" and the "Technical Guiding Principles for the Use of Real-World Data in Clinical Evaluation of Medical Devices (Trial)" have been successively issued, explicitly stating that real-world evidence can be used as relevant evidence for the review and approval of drugs and medical devices. Currently, 13 pilot products utilizing real-world data from Lecheng to assist in clinical evaluation have been approved for marketing by the National Medical Products Administration, continuously bringing "Hainan blessings" to patients across China.

Sanofi Greater China President Wang Shi said:"China continues to deepen the reform of the drug review and approval system. Innovative exploratory initiatives, such as real-world studies in Boao Lecheng, Hainan, have become accelerators for breakthrough innovative drugs to benefit Chinese patients, which is highly encouraging. Riding the wave of favorable policies, Sanofi is actively responding to the call for healthy aging planning and cancer prevention and treatment in Healthy China. We place great emphasis on the unmet needs of hematology and oncology patients and hope to accelerate the introduction of innovative treatments for multiple myeloma into China through real-world study practices. This year, Sanofi has received approval in China for ten innovative drugs and vaccines, including new products and indications. We are advancing the launch of innovative products at an unprecedented speed and will actively explore diverse innovative models to help patients regain the brilliance of life."

Multiple Myeloma is a highly prevalent cancer among the elderly and the second most common hematological malignancy. In China, there are approximately 1.6 multiple myeloma patients per 100,000 people, with the majority of cases occurring in individuals over 60 years old, and the incidence rate is on the rise.^[1]Against the backdrop of China's imminent entry into a moderately aging society and its rapid progression towards a severely aging society, multiple myeloma may impose a greater disease burden.China"Implementing the National Strategy to Actively Address Population Aging" up to nowYear NotThe frequently mentioned topic of healthy aging reflects the urgent need to address the health challenges of "aging," and innovative treatments for multiple myeloma can become an important part of promoting healthy aging.

At the same time, multiple myeloma is still an incurable malignant hematological tumor, and patients will eventually face the dilemma of recurrence.^[2]. Clinically, the more times a patient relapses, the greater the difficulty of treatment, and the shorter the progression-free survival and post-relapse survival time.^[3],[4]Patients urgently need more innovative drugs to expand treatment possibilities. According to the research findings of China's first "Quality of Life Survey Report for Multiple Myeloma Patients," nearly 50% of patients indicated that current treatments fail to meet their expectations regarding overall treatment efficacy and survival benefits, and they hold high hopes for new treatment methods.

Currently, Isatuximab Injection has been approved in the United States, the European Union, the Asia-Pacific region, and many other countries and regions worldwide. Meanwhile, Sanofi is conducting Phase III clinical trials to further evaluate the efficacy of Isatuximab Injection combined with the current standard treatment regimen for multiple myeloma. Research on the use of Isatuximab Injection for treating other hematologic malignancies and solid tumors is also underway.

^[1] Liu J, Liu W, Mi L, et al. Incidence and mortality of multiple myeloma in China, 2006–2016: an analysis of the Global Burden of Disease Study 2016. J Hematol Oncol 12, 136 (2019).

^[2]Plasma Cell Disorders Group of Hematology Branch of Chinese Medical Association, Multiple Myeloma Professional Committee of Chinese Medical Doctor Association. Guidelines for the Diagnosis and Treatment of the First Relapse of Multiple Myeloma in China (2022 Edition) [J]. Chinese Journal of Hematology, 2022, 43(10): 810-817.

DOI:10.3760/cma.j.issn.0253-2727.2022.10.003

^[3] Jagannath S , Roy A , Kish J ,et al. Real-world treatment patterns and associated progression-free survival in relapsed/refractory multiple myeloma among US community oncology practices[J]. Expert Rev Hematol, 2016,9(7):707-717. doi: 10.1080/17474086.2016.1195254 .

^[4] Verelst S , Blommestein HM , De Groot S ,et al. Long-term Outcomes in Patients With Multiple Myeloma: A Retrospective Analysis of the Dutch Population-based HAematological Registry for Observational Studies （PHAROS）[J]. Hemasphere, 2018,2(4):e45. doi: 10.1097/HS9.0000000000000045 .