The year 2024 has been a year full of hope and breakthroughs for China's pharmaceutical field. The emergence of numerous new drugs not only reflects the continuous enhancement of pharmaceutical R&D capabilities but also brings a new ray of hope to a vast number of patients. This year, from tumor treatment to the conquest of rare diseases, and from innovations in traditional drug types to explorations of novel therapies, the development of new drugs has shown a trend of diversification and efficiency. To gain a deeper understanding of the current status of new drug innovation in China, this issue summarizes the information on new drug applications approved by National Medical Products Administration in 2024. By organizing the types of drugs and therapeutic areas, we aim to provide a reference for researchers in the field of biomedical innovation.

Overview of New Drug Approvals in 2024

 Based on the drug approval documents issued by the National Medical Products Administration (NMPA) and the public information on marketed drugs from the Center for Drug Evaluation (CDE) of NMPA [1-2], as of December 31, 2024, a total of 220 new drugs (excluding in vitro diagnostic reagents, vaccines, biological analogues, and other biological products) have been approved by NMPA in 2024. These include 105 new drugs made in China and 115 imported new drugs. Among them, the number of approved pharmaceutical chemicals, therapeutic biological products, and traditional Chinese medicines are 118, 90, and 12, respectively (Figure 1), accounting for 53.6%, 40.9%, and 5.5% of the total number of new drug approvals in 2024.

Approval Status of Class 1 Innovative Drugs in 2024

 Class 1 innovative drugs refer to innovative drugs that have not been marketed inside and outside China. These drugs contain new, structurally defined compounds with pharmacological effects and possess high levels of innovation, clinical value, and social benefits. Therefore, the development of Class 1 innovative drugs also represents a significant shift from exploratory innovation to innovation-driven development.

Class 1 innovative drugs refer to drugs that have not been marketed inside and outside China and belong to the first category in the drug registration classification. These drugs include traditional Chinese medicines and natural medicines, biological products, and pharmaceutical chemicals. In addition, the first-in-class typically feature novel molecular structures with clear pharmacological effects and clinical value. Specifically, Class 1 innovative drugs have the following characteristics:

• APIs and their preparations obtained through synthesis or semi-synthesis methods;

• New effective monomers and their preparations extracted from natural substances or through fermentation;

• Optical isomers of known drugs and their preparations obtained through

•  resolution or synthesis methods;

•  Drugs prepared from multi-component drugs that are already on the market into fewer components;

• New compound formulations;

•  New indications added to marketed preparations in China that have not been approved inside and outside China.

It can be seen that the R&D of Class 1 innovative drugs usually goes through multiple stages, including the discovery phase, preclinical research, clinical trials, and others. Only after being reviewed and approved by the drug regulatory authority can they be marketed. 

Based on data from the Pharma ONE drug R&D big data platform, China National Pharmaceutical Industry Information Center, and NMPA's published drug approval documents (calculated based on approved Class 1 or 1.1 new drugs) [3-4], NMPA approved a total of 46 Class 1 innovative drugs for marketing in 2024, including 7 imported new drugs and 39 new drugs made in China (Figure 2). In terms of drug types, these approvals included 23 pharmaceutical chemicals, 20 therapeutic biological products, and 3 traditional Chinese medicines and natural products, marking an increase of 6 types compared with 2023 and reaching a record in number!

In addition, in terms of therapeutic fields, anti-tumor drugs still topped the list among the Class 1 innovative drugs approved by NMPA in 2024, accounting for 50% (23/46). Unlike the previous two years, drugs for the digestive tract and metabolism ranked second in 2024, accounting for 13.04% (6/46). This was followed by drugs for the nervous system, accounting for 8.70% (4/46). Anti-infective drugs, traditional Chinese medicines, and miscellaneous categories each had 3 approvals, accounting

2024 Annual Approval Status of Anti-tumor Drugs

The continuous emergence of new drugs in the tumor field in China in 2024 is the result of the unremitting efforts of medical researchers. These new drugs play a crucial role in the treatment of various types of tumors, providing more treatment options and hope for survival to tumor patients. Below is a review of some of the new anti-tumor drugs (including their indications and mechanisms of action).

Zevorcabtagene Autoleucel Injection

Indication

For the treatment of adult patients with relapsed or refractory multiple myeloma who have progressed after at least 3 prior lines of therapy (including at least one proteasome inhibitor and one immunoregulator).

Mechanism of action

Zevorcabtagene Autoleucel is a BCMA-targeted CAR-T cell therapy product. CAR-T cell therapy utilizes gene engineering technologies to modify the patient's own T cells, enabling them to express chimeric antigen receptors (CARs) that specifically recognize tumor antigens (in this case, BCMA). These modified CAR-T cells can precisely identify and bind to BCMA on the surface of multiple myeloma cells, activating the T cells' killing function and effectively eliminating tumor cells. This therapy provides an innovative and targeted treatment option for patients with multiple myeloma, especially for those who have experienced poor therapeutic effects with traditional treatments.

Tunlametinib Capsules

Indication

For the treatment of patients with NRAS-mutant advanced melanoma who have failed anti-PD-1/PD-L1 therapy.

Mechanism of action

Tunlametinib is a novel ATP non-competitive MEK inhibitor targeting NRAS mutations. MEK is a key kinase within cells, playing a crucial role in the RAS-RAF-MEK-ERK signaling pathway. In NRAS-mutant melanoma cells, this signaling pathway is often abnormally activated, enhancing the cells' proliferation, survival, and metastasis. Tunlametinib inhibits the activity of MEK, blocking the transmission of this signaling pathway, thereby inhibiting the growth and proliferation of melanoma cells, inducing cell apoptosis, and exerting a therapeutic effect on patients with NRAS-mutant advanced melanoma who have failed anti-PD-1/PD-L1 therapy.

Unecritinib Capsules

Indication

For the treatment of adult patients with ROS1-positive locally advanced or metastatic NSCLC.

Mechanism of action

Unecritinib is a small-molecule drug that specifically targets ROS1-positive NSCLC cells. Its mechanism of action may involve inhibiting the activity of ROS1 kinase and blocking ROS1-related signaling pathways. In cancer cells, abnormal activation of ROS1 promotes malignant behaviors such as cell proliferation, survival, and metastasis. By inhibiting ROS1 kinase, Unecritinib can effectively inhibit the growth of cancer cells, thereby exerting a therapeutic effect on patients with ROS1-positive NSCLC. In the related Phase II research, 111 patients achieved an objective response rate (ORR) of 81.08% based on Independent Review Committee (IRC) assessment, with a median duration of response (DOR) of 20.3 months, demonstrating its efficacy in treatment.

Entinostat Tablets

Indication

For use in combination with aromatase inhibitors in the treatment of patients with locally advanced or metastatic breast cancer who are HR+/HER2- and have experienced recurrence or progression on endocrine therapy.

Mechanism of action

Entinostat is a novel, orally administered HDAC inhibitor. HDACs play a crucial role in regulating gene expression, cell proliferation, differentiation, and apoptosis. In breast cancer cells, particularly HR+/HER2- breast cancer cells, abnormal expression or activity changes of HDACs are associated with tumorigenesis, progression, and resistance to endocrine therapy. By inhibiting HDAC activity, Entinostat can alter the chromatin structure within cancer cells, affecting the expression of related genes, thereby inhibiting cancer cell proliferation, inducing cancer cell apoptosis, and enhancing the efficacy of endocrine therapy. This provides a new treatment option for breast cancer patients who have experienced recurrence or progression on endocrine therapy. In the Phase III clinical research, PFS in the Entinostat group was significantly prolonged compared with the placebo group, with a 24% reduction in the risk of disease progression or death, and a trend towards improved overall survival, with a 16.3% reduction in the risk of death.

Benmelstobart

Indication

For first-line treatment of extensive-stage small cell lung carcinoma (ES-SCLC) in combination with Anlotinib Capsules, Etoposide, and Carboplatin.

Mechanism of action

Benmelstobart is an innovative humanized monoclonal antibody targeting PD-L1 with a novel sequence. In the tumor microenvironment, tumor cells express PD-L1 protein, which can bind to PD-1 protein on the surface of T cells, thereby inhibiting T cell activity and preventing T cells from effectively recognizing and killing tumor cells. Benmelstobart specifically binds to PD-L1, blocking the interaction between PD-L1 and PD-1, and reactivating the immune activity of T cells, enabling them to exert a killing effect on tumor cells. It plays an important role in the combination therapy for ES-SCLC. In the Phase III ETER7011 clinical research, the median OS of patients receiving Benmelstobart combination therapy was 19.3 months, which was 7.4 months longer than that of the chemotherapy-alone group.

Repotrectinib Capsules

Indication

For the treatment of adult patients with locally advanced or metastatic NSCLC that is ROS1-positive.

Mechanism of action

Repotrectinib is a targeted inhibitor of ROS1 and NTRK. ROS1 and NTRK genes are fused or mutated abnormally in some NSCLC patients, leading to abnormal activation of intracellular signaling pathways and promoting malignant behaviors such as cancer cell proliferation, survival, and metastasis. By specifically inhibiting the activity of ROS1 and NTRK, Repotrectinib blocks their related signaling pathways, thereby inhibiting the growth of cancer cells and providing treatment for ROS1-positive NSCLC patients. In the clinical research of adult patients with ROS1-positive NSCLC, for the Chinese subgroup of ROS1 TKI-na?ve patients, the confirmed ORR assessed by BICR reached 91%.

Rezivertinib Mesylate Capsules

Indication

For the treatment of adult patients with locally advanced or metastatic NSCLC who have progressed on or after EGFR-TKI therapy and have been confirmed to have the EGFRT790M mutation by testing.

Mechanism of action

Rezivertinib is a small molecule EGFR kinase inhibitor drug. In NSCLC, EGFR gene mutations are common, especially the T790M mutation, which can lead to resistance to EGFR-TKI drugs. Rezivertinib specifically inhibits the activity of EGFR kinase, particularly in the case of the EGFRT790M mutation. By inhibiting EGFR kinase, it blocks the signaling pathways related to EGFR in cancer cells, thereby inhibiting the growth and proliferation of cancer cells. It provides an effective treatment option for NSCLC patients who have progressed on EGFR-TKI therapy and have the EGFRT790M mutation. In the Phase IIb clinical research for second-line treatment of EGFRT790M-mutated NSCLC, Rezivertinib demonstrated clinical benefits in patients with brain metastases, with a CNSORSummaryR of 69% and a CNDCR of 100%.

The year 2024 has been fruitful for the field of new drugs in China, with a steady increase in the number of new drugs approved. In the tumor field, National Medical Products Administration (NMPA) has approved multiple new anti-tumor drugs, bringing new hope to more patients and providing more treatment options across various types of tumor. According to the data from the Center for Drug Evaluation (CDE) of NMPA, the application for various types of drugs is active. Pharmaceutical chemicals account for the highest proportion (53.6%), followed by therapeutic biological products (40.9%) and traditional Chinese medicines (5.5%). In addition, new drugs that are already marketed abroad but not yet in China are accelerating their entry into the Chinese market, and the phenomenon of a cluster of drugs in the anti-tumor treatment field has shifted. Looking ahead, with the continuous introduction of policies encouraging innovative drug development in China, the gradual improvement of the drug review and approval mechanism, and the continuous enhancement of pharmaceutical companies' R&D capabilities, the environment for innovative drug R&D will further improve. Overall, the application for registration of multiple new drugs in 2024 further testifies to the vigorous development of the pharmaceutical industry and the unremitting pursuit of scientific researchers!

Author's pseudonym (Sophia): Mainly engaged in research on the development of the biopharmaceutical industry and drug popularization.

References：

[1] NMPA, https://www.nmpa.gov.cn/zwfw/zwfwpjfbzs/pjfbzsyp/index.html

[2] NMPA CDE, https://www.cde.org.cn/main/xxgk/listpage/b40868b5e21c038a6aa8b4319d21b07d

[3] Pharma ONE Big Data Platform for Drug Research and Development, one.pharmadl.com

[4] China Pharmaceutical Industry Information Center, https://www.cphiic.com/