NMPA 2021 Drug Approval Review

The National Medical Products Administration (NMPA) in China approved a wide variety of new drugs in 2021, while overcoming a number of challenges in responding to the COVID-19 pandemic. Of note, among the 64 new drugs approved, 28 are domestically developed drugs, and this is a clear sign that the home-grown innovation in China is rapidly advancing.

This report takes a close look at the characteristics of the approved drugs, aiming to provide insights into the development of China biopharma industry, with an emphasis on how biotech and pharma partnerships could further bring significant value to drug development in China.

Part 1: 2021 NMPA Approved Drug  Review

a. Domestic players consolidate strong positions

The incentive policy for innovative drug development in recent years has greatly encouraged the domestic players. Among 64 newly approved drugs (excluding TCM, new indications and vaccines) in 2021, 28 (43.8%) were domestic innovative drugs, reaching a record high in the past four years.

b. 17 (26.6%) new drugs approved in 2021 have already been successfully listed on NRDL

The 2021 National Reimbursement Drug List (NRDL), which was implemented on 1 January 2022, includes 17 new NMPA approvals in 2021 (Table 2). Among the 17 new approvals covered by NRDL, 10 of them are first-time approvals worldwide and developed by domestic companies either through in-house research or partnership. Overall, the updated NRDL list shows China’s strong support and prioritization of innovative drugs, and commitment to bring innovative drugs to a broad population with unprecedented speed.

c. Oncology maintains its leading role

Among the 64 new approvals, 41 are chemical drugs, 21 are biologics and 2 for cell-based therapy. In terms of therapeutic area, oncology, endocrine, infection diseases make up the top 3, similar to previous years. Notably, development in rare disease drugs gains significant traction, and contributes to 13% of drug approval.  

In comparison, FDA approved 50 novel drugs in 2021, with 52% for rare diseases, and 30% for oncology.

Part 2: Highlights

a. Creating value through cross-border partnership  

Demand for innovative medicines has increased substantially over the last five years, yet most of the innovative drugs are only available outside of China.

The in-licensing strategy is considered a highly efficient way for domestic pharma companies to quickly enrich their pipelines and accelerate the path to commercialization. China biopharma continues to source innovations from the global market, leverage its own development capability, and successfully secured 8 such drug approvals in 2021 (Table 3), covering broad therapeutic areas, including oncology, rare disease, and infection diseases.  

b. 77% of approved rare disease drugs are imported

Rare disease drugs are well represented on the list of newly approved therapies. The approval of 9 drugs for rare diseases brings hope to patients who had limited, if not none, available therapies in the past (Table 4).

Of the 9 approvals, 7 are imported drugs, with the exceptions of Herombopag Olamine and Siltuximab. Herombopag Olamine is an innovation drug developed by Hengrui Medicine, a top domestic pharma in China. Siltuximab was co-developed by BeiGene and EUSA Pharma, for the treatment of Multicentric Castleman’s Disease, which again illustrates the value of cross-border collaborations.

In 2021, Basic Health Insurance (BHI) plans in China began to cover the cost of most rare disease drugs. Under the plan, rare disease drugs that are proven effective and that are not excessively priced will be subject to review by experts. The drugs approved after the review will be listed in the NDRL and will be eligible for reimbursement coverage by BHI and other insurance plans.

With all these ongoing mandates, many industry analysts predict that there will be more openings for licensing deals in such domain.   

c. First CAR-T therapy launch in China

Axicabtagene Ciloleucel, FKC876, is an autologous CD19-directed CAR-T cell therapy manufactured in China with the technology of YESCARTA® (Axicabtagene Ciloleucel) from Kite Pharma, a Gilead Company.

The NDA filing of FKC976 is based on results of a single-arm, open label, multi-center bridging trial (FKC876-2018-001) which evaluated the efficacy and safety of FKC876 in the treatment of patients with refractory intermediate invasive non-Hodgkin's lymphoma (NHL)/ large B-cell lymphoma in China.

The approval of FKC876 represents a milestone for cell-based therapy in China and gives Chinese companies confidence to further invest in and bring more world-leading cell therapies to China.

d. Domestically developed ADC goes abroad

Disitamab Vedotin (RC48), a novel ADC developed by RemeGen, was accepted by NMPA in July 2021 for metastatic urothelial cancer. It is the first domestically developed ADC in China to receive marketing approval.

The novel antibody component of the ADC exhibits a higher affinity and increased internalization rate as compared to trastuzumab in preclinical models. In addition, Disitamab Vedotin has shown significant antitumor activity in clinical trials of a number of HER2-expressing cancers, including those with low HER2 expression.

In September 2021, RemeGen and Seagen announced exclusive worldwide license and co-development agreement for RC48, with $200 million upfront and up to $2.4 billion in milestone payments, as well as royalties if approved. For Seagen, the RemeGen deal will allow it to directly challenge breast cancer treatments from Roche and AstraZeneca/Daiichi Sankyo. This out-licensing deal represented a major breakthrough of domestic innovation drug development. Chinese biopharmas are increasingly exploring options to go global and secure the full value of their innovation.

e. The notable gap between global (ex-China) and China launch date for high profile innovative drugs is closing

In 2021, the first domestically developed CDK4/6 inhibitor finally got approval, with 6-year gap comparing to the global launch (In 2015, Palbociclib became the first CDK4/6 inhibitor to receive FDA approval). However, it is exciting to see that the launch lags for Avapritinib and Ripretinib is now less than one year, and both drugs are brought to China market through in-licensing strategy.

Part 3: New drug development in China outlook in 2022

a. MNCs’ China strategy may shift away from Oncology

Although nearly half of approved new drugs by MNCs are oncology drugs, MNCs are expected to invest more in other sectors, rare diseases for example. When challenged by Tagrisso sales in China, AstraZeneca signaled that more efforts will be placed on rare diseases.

It is imperative for MNCs to achieve success in China by diversifying their portfolio. Some domestical companies may follow the similar portfolio strategy, but others may start finding their own niche and exploring advantages by tackling overlooked therapeutical areas.

b. Rising innovation from Chinese biopharma

Domestic companies see innovation as a must for maintaining sustainable growth. Taking BeiGene as one example, it launched four innovative drugs (Carfilzomib, Pamiparib, Stauroximab and Daltosimab Beta), via both in-house development and external partnership strategy. With the streamlining of the drug commercialization process, Beigene’s future development momentum looks quite strong.

c. Novel biotechnology gains momentum

Novel technologies such as cell-based therapy, protein degrader, xRNA therapy, and microbiome have already begun to gain more traction. Leading companies will look to further diversify their pipeline and turn to the global market for innovative technology platforms collaborations.

d. Growing demands for early-stage collaborations

Driven by strong economic growth and health demands of an aging population, China’s pharmaceutical market is projected to grow substantially. Thus, China market will continue to attract MNCs such as AstraZeneca, Roche, Merck, Sanofi, and Novartis. The collaboration between MNCs and local players is on the rise, which will create tremendous value for both parties.

It is also noteworthy that collaboration between domestic companies is another emerging trend. The partnership between Innovent and IASO Biotherapeutics (BCMA CAR-T), and the one between Beigene and Nanjing Leads Biolabs (Lag3 antibody) are such examples.  It is true that while China’s own innovation engine is starting to take shape, much of its focus is directed towards validated targets and modalities. Given that it is difficult to compete with MNCs on the truly innovative programs that are already in late-stage development, one option for domestical companies is to turn towards novel assets that are class leading and early in development, which necessitates starting collaboration with ex-China counter parties at early stage and aim for a good return on long term investment.

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