The regulatory landscape of advanced therapies in Asia-Pacific

Feature ArticlesFeature Articles
| 31 October 2021 | By Ellen Sem, BS (Pharmacy), RACVicky Han, BPharm, MBAAnnetta C. Beauregard, MS, MBA  | ©

The Asia-Pacific region, which inlcudes Australia, China, Hong Kong, Japan, Malaysia, Singapore, and South Korea, has a significant number of clinical trials in advanced therapies, with China ranking second globally. Manufacturers in other countries in the region, such as Japan, Australia, South Korea, and Singapore, have submitted marketing authorizations applications for advanced therapies and received approvals. Regulators recognize this emerging trend in dynamic research and development and have started developing regulatory frameworks and pathways to accommodate it. This article will look at the regulatory landscape for advanced therapies in different markets within the Asia-Pacific region and the marketing authorization procedures available in these markets in the absence of a harmonized regulatory pathway.
Advanced therapies (also known as human cell, tissue, and gene therapy products or regenerative medicinal products) hold the promise to treat medical conditions and diseases that currently have limited or no effective therapeutic options. There is currently no unified terminology for this category of product. For this article, the term advanced therapies will be used and include viable autologous or allogeneic human cells or tissues, viable xenogeneic cells and tissues, and gene therapy products. According to the Alliance of Regenerative Medicine H1 2020 Report,1 there are currently 1,078 phase 1 to phase 3 ongoing clinical trials worldwide, with a majority in the phase 2 stage. Cell-based immune-oncology has the highest number products in the pipeline, although gene therapy has the highest number of phase 3 studies. Following approval of the first two chimeric antigen receptor T-cell (CAR-T) therapies, Kymriah (tisagenlecleu-cel) and Yescarta (axicabtagene ciloleucel), in the EU2 and US,3 they have also been approved in the Asia-Pacific countries of Australia4 and Japan5 over the past 2 years. Zolgensma (onasemnogene abeparvovec) was approved in Japan in March 2020.
As of July 2021, the region has seen more advanced therapies approvals in China, Japan, South Korea, Singapore, and the Hong Kong markets, including:
  • March 2021, Kymriah approved in South Korea6 and Singapore;7
  • June 2021, YiKaida (also known as Yescarta), which was the first CAR-T cell therapy approved in China8 under priority review;
  • March 2021, Breyanzi approved in Japan;9
  • May 2021, Zolgensma approved in South Korea;10 and
  • June 2021, Kymriah approved in Hong Kong.11

This rapid growth in research and development of advanced therapies in Asia-Pacific has spurred regulatory agencies to develop a regulatory framework for facilitating accelerated marketing authorizations for advanced therapies to meet unmet medical needs. This article aims to summarize the current state of the regulatory frameworks in several markets in Asia-Pacific namely, Australia, China, Hong Kong, Japan, Malaysia, Singapore, and South Korea, 


Advanced therapies are classified under therapeutic products in the Therapeutic Goods Administration (TGA), Australia’s regulatory authority. Human cell and tissue therapy products (e.g., products in tissue banks, stem cell therapy products, and gene-modified cell therapies) are classified as biologicals.12 The Australian regulatory framework for biologicals13 provides the legislative basis for the regulation of human tissue and cell-derived products supplied in Australia. The framework applies different levels of regulation to products based on the risks associated with their use. It is designed to be flexible enough to accommodate emerging technologies. There are four classes of biologicals (Class 1, 2, 3, and 4) in Australia and they are stratified by risk-based approach:
  • Class 1 biologicals are low risk and have an appropriate level of external governance and clinical oversight.
  • Class 2 biologicals are low risk and have been subjected to only manipulation and are only for homologous use; they include human ocular and cardiovascular tissue and amniotic membrane.
  • Class 3 biologicals are medium risk, for homologous use but have been prepared using more than minimal manipulation or for nonhomologous use, regardless of whether they have been prepared using minimal manipulation or more than minimal manipulation, which includes demineralized bone matrix.
  • Class 4 biologicals are high-risk products that are more than minimally manipulated to artificially introduce a function of the cells or tissues that are are not intrinsic to the original cells or tissues; or pluripotent stem cells; or biologicals derived from pluripotent stem cells.

Most human cell and tissue (HCT) therapy products will be regulated under Classes 2, 3, and 4.
As CAR T-cell therapy involves genetic modification of cells and the intrinsic function of the donor biological has been changed, it is classified as a Class 4 biological. The level of dossier requirements to demonstrate of efficacy, safety, and quality will depend on the level of classification. The regulatory pathway for in vivo gene therapy products (e.g., adeno-associated virus vectors) are classified as prescription medicines14 in which the sponsor will need to fulfil the requirements stipulated in the Australian Regulatory Guidelines for Prescription Medicines.15
Priority review and provisional approval are fast track approval pathways that allow certain prescription medicines for serious or life-threatening conditions accelerated access into the Australian market. For provisional approval pathway, the sponsor can apply for time-limited provisional registration for certain promising new medicines, based on preliminary clinical data where there is the potential for a substantial benefit to Australian patients. Provisional registration of prescription medicines under this pathway is limited to a maximum of 6 years.16 This pathway could make a product available for use up to 2 years earlier than normal. For priority review, TGA can complete the review up to 3 months earlier because of the more flexible evaluation process. Priority review involves the same amount and type of evidence as the standard review process.17
TGA has also adopted international guidance documents – for example, European Medicines Agency (EMA) guidelines on quality, safety, and efficacy aspects in cell and gene therapy manufacturing – which are published on the TGA’s International Scientific Guidelines followed in Australia.18 Some autologous human and cell tissue products19 may be excluded from TGA regulation or exempt from some TGA regulations. The level of regulation for autologous human and cell tissue products is based on the level of risk, level of external governance and clinical oversight, or depending on the manufacturing processes or intended use of the autologous HCT product. In May 2021, the TGA conducted a public consultation seeking feedback on a proposal to remake some of the legislative instruments relating to HCT products (including blood and blood components), which sunset in October 2021.20
Genetically modified organisms (GMOs) are also regulated by the Gene Technology Act 2000.21 The law was established to protect public health and safety and the environment by identifying risks posed by, or because of, gene technology and by managing those risks through regulating certain dealings with GMOs that include gene therapy products and certain Class 4 biologicals. The Office of the Gene Technology Regulatory (OTGR)22 is responsible for providing the regulatory oversight for the law, and applicants must apply to OTGR to handle or import such products in Australia.


In 2003, the world’s first gene therapy product, Gendicine (recombinant human p53 adenovirus) for treating advanced head and neck cancer, was approved in China.23 China ranks second in the global advanced therapies clinical trials map in a Deloitte 2020 analysis.24 The National Medical Products Administration (NMPA) is the regulatory agency that provides the regulatory oversight of advanced therapies. In recent years, the agency has promulgated many guidelines to govern the research and development of advances therapies:
  • 2015, guidelines for managing stem cell clinical research, stem cell preparation quality control, and preclinical research (issued by the National Health and Family Planning Committee and China Food and Drug Administration, NMPA’s predecessor);
  • 2017, guiding principles for cell therapy product research and evaluation;
  • 2018, guideline on chemistry, manufacturing, and controls (CMC) study and application document requirements for cell therapy clinical trial applications;25
  • Since 2019, NMPA has launched the China Drug Regulatory Scientific Action Plan,26 which includes cell and gene therapy products as a first batch of priority projects. Through this action plan, the agency has published a series of draft guidelines for industry and academia consultation to improve the evaluation process of advanced therapies for industry and academia consultation. These guidelines cover a range of topics (e.g., CMC, nonclinical, gene transduction, clinical trials conduct) in cell and gene therapy;
  • In February 2021, NMPA finalized a guideline for clinical trials for immunocyte therapy products;27
  • In June 2021, the agency issued a public consultation for a new draft guideline on the requirements for clinical studies with long-term follow-up of gene therapy products.28

Under the existing framework, cell and gene therapy products are classified as biological products and sponsors would need to fulfill the requirements for a biological product in China. Early consultation with the agency to discuss the clinical development plan is strongly encouraged. In 2020, the NMPA published the New Drug Registration Regulation, which established the four expedited review pathways for drugs used in serious life-threatening diseases to fulfill local unmet medical need: breakthrough therapy designation, conditional approval, priority review, and special emergency approval for public health emergencies.29 Most advanced therapies would qualify for these expedited pathways, which allow for quicker research and development and eventually faster product approvals.
The agency has also taken an active role in the international arena and submitted two reflection paper proposals to the International Council for Harmonisation (ICH) on cell and gene therapy product,30 which demonstrated its commitment to advocate international cooperation and harmonization in advanced therapies. In the most recent rendition of China’s 5-year plan and goals for national economic and social development, published in March 2021,31 the government has prioritized the innovation of gene and regenerative medicine to encourage research and development in advanced therapies. The hope is that having strong policy in place will provide continued support for the advanced therapy environment and eco system and that it will facilitate more multilateral collaborations between academia, biotech companies, and government.


In Japan, there are two main laws regulating regenerative medical technology and products – The Act on the Safety of Regenerative Medicine and The Act on Pharmaceuticals and Medical Devices (PMD).32 Enacted on 25 November 2014, this was the first-of-its-kind framework regulating regenerative medicine in Asia-Pacific.32 In June 2014, the Ministry of Health, Labour and Welfare (MHLW) announced the SAKIGAKE strategy for the practical application of innovative medical products. One of the major components of the strategy is its designation system, which promotes early research and development and clinical trials by conducting priority consultations, early assessment, and priority reviews for early phase innovative medical products including regenerative medicine that have potential efficacious results.
The law on the safety of regenerative medicine provides regulatory oversight on all medical technologies using processed cells in medical care or academic research purpose, whereas the PMD Act regulates the marketing authorization and commercialization of a regenerative medicinal product. Under this framework, regenerative medical products include both cellular and tissue-based products to be used in the reconstruction, repair, or formation of structures or functions of the human body and the treatment or prevention of human diseases, and gene therapy.
The Pharmaceutical and Medical Devices Agency (PMDA) created a special pathway that allows applicants to seek conditional/time-limited approval with phase 2 study data (after the compound has confirmatory safety and probable efficacy data). The conditional approval is valid for 7 years maximum. The applicant will need to submit a subsequent application to PMDA to provide confirmatory efficacy and safety data in which PMDA will assess the data, before granting the full marketing authorization approval.
In addition to the conditional approval pathway, the sponsor of a regenerative medicine can also apply for the Sakigake designation. To qualify, the drug must be firstly developed in Japan and planned an application for approval, innovative, meant for life-threatening diseases with prominent efficacy. Benefits for Sakigake designation include prioritized consultation, de facto review before application, priority review time reduced from 12 months (standard evaluation time) to 6 months, extension of re-examination period up to 10 years, working with a dedicated PMDA project manager.34 There were 11 regenerative medicines with Sakigake designation35 from February 2016 to April 2019.
Over the years, the PMDA has also issued guidelines for different types of regenerative medicine, including guidelines on ensuring quality and safety of gene therapy products36 and on cancer immu­notherapy development.37 The agency launched regulatory science strategy consultations to provide advice on early development trials and the requirements for starting clinical trials in Japan. For regenerative medical products, regulatory science strategy consultations on quality (e.g., viral safety, microbial contamination, specification) and safety (e.g., general toxicity, tumorigenicity if applicable) is mandatory for initiating a clinical trial.
Gene therapy products (in vivo and ex vivo) in Japan are regulated by the Cartagena Act on the Conservation and Sustainable Use of Biological Diversity Through Regulations on the Use of Living Modified Organisms (LMOs). This law concerns the use of LMOs in Japan. Prior approval by the MHLW and Minister of the Environment is required when attempting Type 1 use to ensure the LMOs do not have negative effect on the local environment. It is necessary for the sponsor to apply and obtain the approval of the MHLW regarding the Type 1 use at the medical institution before starting the clinical trial.38
As the pioneer of regenerative medicine, Japan is expected to grow as an investment and approval destination for multinationals developing regenerative treatments. In March 2020, the government of Japan approved the second phase of its 5-year health and medical strategy,39 which was implemented on 1 April 2020. The new strategy established six priority areas for further research and development, including pharmaceuticals; regenerative, cell, and gene therapies; genomes and data platforms; medical equipment; and basic disease research.39


South Korea
South Korea has an active research and development environment in advanced therapeutics. As of July 2019, there were 16 cell therapies approved in South Korea, all developed locally and approved under the Pharmaceutical Affairs Act, which provides the regulatory framework for advanced therapies before 2021.
In 2020, the South Korea Ministry of Health and Welfare (MoHW) brought the new Advanced Regenerative-Bio Act40 into effect to improve access to advanced regenerative medicine and the advanced biopharmaceuticals products in South Korean market. The new act will provide the basis for a whole life-cycle safety management system of advanced regenerative medicine and advanced biopharmaceuticals using stem cells and technical development in the fast-growing market of regenerative medicine. In this new framework, MoHW has regulatory oversight of medical practice and clinical research use of regenerative medicine, whereas the Ministry of Food and Drug Safety (MFDS) is overseeing the commercial use and manufacturing of cell and gene therapy.
Key changes in the new act41 include:
  • Definitions and the scope of advanced regenerative medicine and human cell therapy (human cell therapy, gene therapy, tissue engineering and fusion therapy are classified as advanced regenerative medicine);
  • A dedicated expediated procedure that allow faster evaluation;
  • A customized review system that allows for prior screening of documents in different phases in line with the sponsor’s timeline; and
  • Conditional approval in which sponsors can apply for using phase 2 clinical data to demonstrate efficacy and safety and supplement with phase 3 or confirmatory clinical data.

With this new act, the industry anticipates shorter research and development time for these products of up to 4 years. The ministry will designate the advanced biopharmaceuticals as products for fast track since they should be developed swiftly. The commercialization of the products will be supported by customized and priority reviews from the early stage of development. The approval process of the products for fast track is shortened from 115 days to 90 days.43 MFDS has also issued various guidelines in cell, gene, and tissue therapy, such as guidelines on quality assessment for gene-editing based advanced therapy medicinal products,4 nonclinical assessment of gene therapy products,45 and cell-based combination products using scaffold. Sponsors will also need to fulfil the requirement stipulated in the guidelines, depending on the product category.
To enhance the competitiveness of local environment in advanced therapies, MHLW also set up the Korea Institute of Drug Safety & Risk Management. The institute serves as a regulatory science center for researching and investigating policies related advanced therapies and nurturing the experts needed for manufacturing and managing the quality of the products.



In Singapore, advanced therapeutics are known as cell, tissue, and gene therapy products (CTGTPs) and are regulated by the Health Sciences Authority (HSA). The Health Products Act (HPA) and Health Products (Cell, Tissue and Gene Therapy Products) Regulations, which came in effect on 1 March 2021, provide the main regulatory framework for CTGTPs.
The products are risk-stratified into Class 1 (low risk) and Class 2 (higher risk).46 To be classified as Class 1, the CTGTP must fulfil all the criteria: minimally manipulated; intended for homologous use and not combined; or used in conjunction with therapeutic products or medical devices and examples will include bone grafts, amniotic membrane. Other CTGTPs not classified as Class 1 will be classified as Class 2, which includes CAR-T cell therapy, gene therapy, and culture-expanded cells. The framework covers the whole lifecycle management of CTGTPs, from clinical trials to product registration, postapproval variations, dealers’ licenses, safety monitoring, and product recall.
As HSA adopts a risk-based approach, the lower-risk Class 1 CTGTP requires only prior notification to HSA. Full product registration is required for Class 2 CTGTPs, and HSA may grant conditional registration with postapproval commitments to a Class 2 CTGTPs that meet all the criteria: intended to treat an unmet medical need; product safety established in early clinical trials; and preliminary data shows meaningful evidence of therapeutic benefit (a clinically significant endpoint) compared with other available therapies (standard of care).47 Results of the confirmatory studies should be submitted within a prescribed timeline for evaluation before obtaining full marketing approval.
Research, innovation, and enterprise are cornerstones of Singapore’s national strategy to develop a knowledge-based, innovation-driven economy and society. Building on the government’s Research, Innovation and Enterprise (RIE) 2020 plan, Singapore government announced in March 2019 that it would be investing S$80 million to develop core capabilities required for cell therapy manufacturing.48 The Agency for Science, Technology and Research Singapore has dedicated teams to conduct cutting-edge discovery research in advanced therapies49 to develop expertise to contribute the development of local eco system.


In Malaysia, advanced therapies are also termed cell and gene therapy products (CGTPs) and are regulated as biologic products by the National Pharmaceutical Regulatory Agency (NPRA). The guidance document and guidelines for registration of cell and gene therapy issued in March 2016 provide the regulatory framework for CTGTPs and implementation of the guideline started on 1 January 2021.50 NPRA adopted a risk-based approach in the CGTP framework with two risk classes – Class I (low risk) and Class II (high risk). For a CGTP to be classified as class I, it needs to meet all the following criteria: minimal manipulation; homologous use; no combination with drug/device/article; no systemic effect and is not dependent upon metabolic activity of living cells for its primary function. Class I CGTPs examples would include bone graft, hematopoietic stem cells. CGTPs that do not meet the criteria for Class I will be classified as Class II, which includes CAR-T cell therapy and novel cell and gene therapies. For Class II CGTPs, the applicant will need to submit a biological license application to NPRA with the necessary quality, efficacy and safety information required by NPRA. Sponsors can also apply for conditional approval and/or priority review for CGTPs in Malaysia. The conditional approval will allow sponsors to submit earlier using early clinical data based on fully validated surrogate endpoints or other relevant early data if the CTGP is used for seriously debilitating diseases or life-threatening diseases or designated as orphan drug.51 For priority review, the timeline can be shortened from 245 working days to 120 working days.52


Hong Kong
In Hong Kong, the Pharmacy and Poisons (Amendment) Ordinance 2020 to regulate advanced therapy was passed by the legislative council and gazette in July 2020.53 Since 1 August 2021, when the amendment ordinance came into effect, an advanced therapy product will fall under the definition of pharmaceutical product and will be regulated as a specific subset of pharmaceutical products in Hong Kong. Under the framework, advanced therapy product will include gene therapy product; a somatic cell therapy product; and a tissue engineered product. The Department of Health has also issued guidance documents for product registration before marketing, prior approval for clinical trials, licensing of the manufacturers and distributors, labelling, record keeping, and import and export control.54 An applicant will need to fulfil the requirements outlined in the guidance to register for an advanced therapy product.


Future regulatory trends in Asia-Pacific
As mentioned in the introduction, the region has seen more early approvals in advanced therapies with the introduction of regulatory frameworks by the regulators. These approvals demonstrated that the regulators in this region are more ready to evaluate and approve the advanced therapies. Many multinational pharmaceutical companies are leveraging on these new regulatory pathways for advanced therapies to bring these innovative medicines into Asia-Pacific. The region continues to be a hot spot for advanced therapies with the various policy support introduced by the governments to foster innovation, research, and development in Asia-Pacific. Beside the proposals submitted by NMPA to ICH in gene therapy, both MHLW/PMDA and NMPA are in the expert working group for the new ICH S12 Guideline on “Nonclinical Biodistribution Considerations for Gene Therapy Product,55 which reflect the emerging trends of advanced therapy in Asia-Pacific. As this space is rapidly evolving, both the regulators and industry need to be agile and flexible to accommodate this novel technology. The regulators mentioned in the article have adopted a risk-based approach and expedited pathways in regulating and evaluation.
This article provides a high-level overview on the regulatory frameworks for advanced therapies available in Asia-Pacific and it is important to know that the Asia-Pacific regulators are monitoring the scientific development closely and will continue to issue new or revise the regulations and guidelines in the future.
AP, Asia-Pacific; CAR, Chimeric antigen receptor; CGTP, cell and gene therapy products; CTGTP, cell, tissue and gene therapy products; DOH, Department of Health; EMA, European Medicines Agency; GMO, Genetically modified organisms; HSA, Health Sciences Authority; ICH, International Council for Harmonization; LMO, Living Modified Organisms; MFDS, Ministry of Food and Drug Safety; MHLW, Ministry of Health, Labour and Welfare; MoHW, Ministry of Health and Welfare; NMPA, National Medicinal Product Administration; NPRA, National Pharmaceutical Regulatory Agency; OTGR, Office of the Gene Technology Regulatory; PMDA, Pharmaceutical and Medical Devices Agency; TGA, Therapeutic Goods Administration
About the authors
Ellen Sem, BS (Pharmacy), RAC, is associate director and Asia-Pacific regulatory policy and intelligence lead at Johnson & Johnson, Singapore, with more than 10 years regional regulatory affairs experiences across multiple therapeutic areas in the pharmaceutical industry. She is also an experienced project manager and has worked on products at various stages of development, from early through to filing, commercialization, and post approval. Sem is an active member of various Asian regional trade associations. She received a bachelor of science degree in from the National University of Singapore and holds certification in regulatory affairs (RAC) and from the Quality Assurance Graduate Program, at Temple University. She can be contacted at
Fengyun (Vicky) Han, BPharm, MBA, is senior director and head of the global regulatory policy and intelligence for Asia-Pacific global regulatory affairs at Janssen. She has more than 25 years regulatory of experience in Asia-Pacific and Europe, encompassing a range of products, including chemical and biological, vaccines, biosimilars, and generics. Han is an active member of many trade associations and has held leadership positions in some. She has a bachelor of pharmacy degree and a master of business administration degree. Han can be contacted at
Annetta C. Beauregard, MS, MBA, is vice president of Janssen Global Regulatory Policy and Intelligence. She is responsible for leading, developing, and driving global regulatory intelligence, policy, and advocacy to advance Janssens’ pipeline for patients. Beauregard has more than 20 years of regulatory policy experience. She has a master of science degree from the University of Indianapolis and a master of business administration degree. She can be contacted at
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