China's CDE Releases Third Batch of Clinically Urgent Overseas New Drugs List Featuring Blockbuster Therapies

Pharmaceutical Network, October 30 — On October 29, the Center for Drug Evaluation (CDE) publicly released the third batch of the List of Overseas New Drugs in Urgent Clinical Need. The list includes seven specialty drugs that are not yet marketed in China and are in urgent clinical need, such as Ablynx’s nanobody drug Cablivi, Astellas’ FLT3 inhibitor Xospata, and Agios’ leukemia drug Tibsovo. These drugs cover therapeutic areas including hematologic disorders, malignant hematologic diseases, genetic metabolic diseases, neurological disorders, infectious diseases, and ocular diseases.

 

 

In October 2018, the nationalPharmaceuticalsthe National Medical Products Administration, in conjunction with the National Health CommissionHealthThe Committee organized the drafting of the “Procedures for the Review and Approval of Overseas New Drugs in Urgent Clinical Need,” establishing a specialized channel to expedite the review and approval of overseas marketed new drugs that are in urgent clinical need. It is reported that the scope of varieties included in the list of overseas marketed new drugs in urgent clinical need primarily comprises new drugs approved for marketing in the United States, the European Union, or Japan in recent years but not yet marketed in China, which are used for the treatment of rare diseases, as well as those used for the prevention and treatment of conditions that are seriously life-threatening or significantly impair quality of life.Disease, and there are currently no effective treatment options or new drugs with significant clinical advantages.

 

Subsequently, the Center for Drug Evaluation (CDE) released two batches of lists of overseas new drugs urgently needed for clinical use in November 2018 and May 2019, covering a total of 66 drugs. According to statistics, as of October 2020, 37 overseas new drugs, including Amgen’s denosumab injection, Merck & Co.’s pembrolizumab injection, and Pfizer’s tafamidis meglumine soft capsules, had all been approved for marketing in China, further alleviating patients’ urgent need for medication.

 

Among the third batch of overseas new drugs urgently needed for clinical use announced in this public notice, three drugs have already submitted marketing applications in China. In April this year, Astellas announced that the NMPA had accepted its New Drug Application (NDA) for Xospata, a once-daily oral therapy, for the treatment of adult patients with relapsed or refractory (treatment-resistant) acute myeloid leukemia (AML) who are FLT3 mutation-positive (FLT3mut+). Currently, Xospata has been officially included in the priority review program, and industry predictions suggest that the drug is expected to be approved for marketing in the second quarter of 2021. Publicly available data shows that Xospata generated $136 million (14.3 billion yen) in sales revenue for Astellas in 2019.

 

Furthermore, Biogen’s new multiple sclerosis drug, dimethyl fumarate enteric-coated capsules (Tecfidera), submitted its marketing application in China this February. In 2019, Biogen achieved $4.4 billion in sales revenue with this blockbuster small-molecule drug. Currently, Tecfidera has entered the first round of supplemental review and is expected to be approved in the first quarter of 2021. Notably, eight domestic pharmaceutical companies, including Jiangxi Qingfeng Pharmaceutical, Shanghai Huilun Jiangsu Pharmaceutical, and Lipin Pharmaceutical, have also begun to lay out their strategies for this product.

 

The third drug that has been submitted for marketing approval in China is the novel super anti-influenza medication baloxavir marboxil tablets (Xofluza). Xofluza was developed by the Japanese pharmaceutical company Shionogi. In 2016, Roche entered into a collaboration agreement with Shionogi to jointly oversee the drug’s development outside Japan and Taiwan, with Roche holding commercialization rights in all regions except Japan and Taiwan. As the first new anti-influenza drug approved in the United States in nearly two decades, Xofluza generated $234 million in revenue for Roche in 2019, leveraging its novel mechanism of action against influenza.

 

 

From the industry’s perspective, recent pharmaceutical regulatory reforms have significantly accelerated the entry of overseas new drugs into the Chinese market, benefiting domestic patients while also bringing about substantial changes to the environment for new drug research and development in China.

 

Analysts have pointed out that the consistency evaluation of generic drugs is driving the internationalization of the quality of China's generic drugs, while the NMPA's accession to the ICH represents the internationalization of regulatory systems. The regulatory authorities' encouragement of multi-regional clinical trials (MRCT) reflects the internationalization of innovative drug research and development, and the accelerated entry of imported drugs into the Chinese market indicates that domesticPharmaceuticalsThe market is becoming increasingly internationalized, and the internationalized industrial ecosystem has an impact on domestic pharmaceuticalsEnterprisepresents entirely new requirements and challenges. “Such environmental conditions require domestic pharmaceutical companies to shift their new drug R&D from ‘China-new’ to ‘global-new,’ and to expand from the Chinese market to the global market.”

 

However, it cannot be ignored that the development of new drugs targeting popular pathways such as PD-1/PD-L1 and BTK inhibitors has already fallen into a red ocean of homogeneous competition. In response to the challenges faced by domestic pharmaceutical companies in innovative drug development, an R&D executive at a pharmaceutical company pointed out that the solution undoubtedly lies in enhancing the innovativeness of new drug development, improving the efficiency and speed of R&D, and accelerating internationalization, among other dimensions.

 

“Developing first-in-class innovative drugs in China currently faces significant challenges, as the R&D foundation of pharmaceutical companies is not yet robust and numerous barriers remain. Nevertheless, it is essential to remain committed to this goal and continue advancing in this direction. Naturally, a higher degree of drug innovation entails greater risks of R&D failure, and success rates vary considerably between different drug categories, such as large-molecule biologics and small-molecule chemical drugs. This necessitates in-depth research by pharmaceutical companies. Achieving differentiation for drugs targeting existing pathways also represents a key breakthrough avenue, including through the development of new indications and innovative drug combinations. Furthermore, an important aspect of the new drug R&D process is enhancing efficiency and speed—while ensuring drug quality—by optimizing processes, adopting new technologies, establishing key technology platforms, and strengthening translational research, thereby building a highly efficient R&D system.”