Eddie Tan, PhD, CABP
ISCT Asia Pacific (APAC) Industry Committee Member
ESP Subcommittee Member
Center of Regulatory Excellence, Duke-NUS Medical School
Singapore

On behalf of the ISCT Asia Pacific (APAC) Industry Committee

Webinar Co-Chairs

• William Hwang, MBBS, FRCP, FAMS, MBA
  ISCT APAC Industry Committee Co-Chair
  Singapore
  
  
• Eddie Tan, PhD, CABP
  ISCT APAC Industry Committee Member
  ESP Subcommittee Member
  Singapore
  
  

Speakers/Panelists

• Jihye Shin, PhD
  Xcell Therapeutics Inc.
  South Korea
  
  
• Sudipto Bari, PhD
  ISCT Asia Regional ESP Subcommittee Co-Chair
  Advanced Cell Therapy and Research Institute (ACTRIS)
  Singapore
  
  
• Yoshiaki Maruyama, PhD
  Pharmaceutical and Medical Devices (PMDA)
  Japan

Regulatory Insights from Japan, Singapore and South Korea

Over the past decade, Asia Pacific has emerged as a global hub for cell and gene therapy innovation. However, the regulatory landscape across this region remains fragmented, with different requirements, timelines, and strategic approaches from market to market. This creates both tremendous opportunities and significant challenges for sponsors, investigators, and clinicians. 

The ISCT APAC Industry Committee had convened three leading regulatory experts who've navigated these pathways firsthand in three of Asia Pacific's most important markets: Japan, Singapore, and South Korea, and they shared critical regulatory insights from their respective markets and the broader implications for pan-Asia strategy.

Regulatory Diversity: A Challenge for CGT Product Developers

Despite this promise, the APAC region presents a complex regulatory environment that poses significant challenges for product developers. Each country has established its own regulatory frameworks, approval processes, and documentation requirements for CGT products. This regulatory patchwork results in a lack of harmonization, making it difficult for developers to navigate clinical trial approvals, manufacturing standards, and post-approval changes across borders. Additional logistical issues, such as import/export restrictions on genetic materials, complex documentation, and varying healthcare provider capabilities, compound these challenges.

Regulatory Frameworks and Market Overview in Asia Pacific Cell and Gene Therapy

Japan's Regulatory Framework for Regenerative Medicine

• Yoshiaki Maruyama, PhD (PMDA, Japan), presented Japan’s dual regulatory scheme for regenerative medicine:
  • The Act on the Safety of Regenerative Medicine (ASRM) governs medical technologies and hospital exemptions, including clinical research.
  • The Pharmaceuticals and Medical Devices Act (PMD Act) regulates commercial medical products.
  • Japan classifies regenerative medical technologies by risk: Class 1 (high risk, e.g., embryonic stem cells, gene-modified cells), Class 2 (non-homologous use), and Class 3 (autologous cells).
  • Japan has approved 21 cell and gene therapy products, mostly targeting orphan diseases, with a standard review period of 12 months (priority review 9 months).
  • Japan uses a conditional and time-limited approval scheme allowing early marketing authorization with post-marketing surveillance, valid up to 7 years with possible extensions.
  • Clinical trial notifications have stabilized at about 15 annually since 2016, with oncology as the largest development area.

Singapore’s Regulatory and Clinical Services Framework

• Sudipto Bari, PhD (ACTRIS, Singapore), outlined Singapore’s regulatory landscape governed by two authorities:
  • Health Sciences Authority (HSA) regulates cell, tissue, and gene therapy products (CTGTPs) under the Health Products Act.
  • Ministry of Health (MOH) regulates clinical services under the Healthcare Services Act.
  • Singapore classifies CTGTPs into Class 1 (low risk, minimal manipulation, homologous use) and Class 2 (moderate to high risk, non-homologous use).
  • Regulatory controls cover clinical trials, manufacturing licenses, product registration, adverse event reporting, and special access routes.
  • Clinical trial applications include notification and authorization pathways, with requirements for process validation and CMC data.
  • Product registration offers full and bridge routes, with the bridge route allowing faster approval (11.5 months) if approved by recognized overseas regulators (e.g., FDA, EMA).
  • Singapore has approved six Class 2 CTGTPs as of March 2025 and supports access to unregistered products under strict controls.
    GMP and GDP guidelines are aligned with international standards (PICS).
  • Singapore has implemented reimbursement frameworks in the public sector and private insurance coverage for approved CTGTPs.
  • Clinical services regulation includes licensing for in-house manufactured non-commercial CTGTPs, with pathways for generally accepted treatment, innovative service therapy, constrained use, and expanded access.

South Korea's Dual Track Regulatory System and Market Strategy

• Jihye Shin, PhD (Xcell Therapeutics Inc., South Korea), described South Korea’s proactive regulatory environment with a dual track system:
  • Track 1: Advanced Regenerative Medicine (ARM) for non-commercial clinical research at designated hospitals.
  • Track 2: Advanced Biological Products (ABP) for commercial product development from IND to BLA.
  • South Korea aims to reduce BLA approval time from 420 to 295 days by 2025, supported by a new Special Act for CDMO sector implementation in 2026.
  • ARM research expanded to all indications in 2024, with risk-based classification (high, medium, low LISC) affecting approval requirements.
  • Developers can charge for ARM therapies during clinical research under new regulations effective February 2025, enabling revenue generation and real-world evidence collection.
  • IND submissions require Korean translations and local representation; BLA review includes priority and conditional approvals, especially for rare and life-threatening diseases.
    
  • The GIFT program offers a fast-track 90-day review with a high approval rate, mainly for rare and severe diseases.
  • Bridging study waivers are possible by including Korean sites in global trials.
  • Long-term follow-up (up to 15 years) is mandatory for all CGT products, integrated from early development stages.
  • Pricing and reimbursement involve risk-sharing agreements to balance patient access and national insurance sustainability.
    

Panel Discussion on Regulatory Trends and Harmonization Moderated by Professor, William Hwang, ISCT APAC Industry Committee Co-Chair

Pending Regulatory Changes and Initiatives

Yoshiaki Maruyama, PhD, reported no immediate pending regulatory changes in Japan but emphasized ongoing updates to adapt to rapid field developments. Shin highlighted the upcoming 2026 Special Act in South Korea, aimed at supporting CDMOs and the 2025 Regenerative Medicine Bio Act enhancing early patient access and funding. Bari noted that Singapore’s regulatory framework is relatively nascent compared to the other two jurisdictions (since 2021) and expressed interest in future harmonization and synergy to accelerate development and access.

Impactful Recent Policy Developments

Japan’s conditional and time-limited approval scheme has significantly impacted CGT oversight, with ongoing discussions about reimbursement under national health insurance. Singapore’s establishment of product regulations and clinical services frameworks since 2021 has laid a foundation for growth and potential harmonization. South Korea’s reforms targeting reduced approval times and expanded access pathways have facilitated market growth and innovation.

Regulatory Openness to Adaptive Designs and Real-World Evidence

The COVID-19 pandemic demonstrated regulators’ agility and increased acceptance of risk-based approaches, adaptive designs, and real-world evidence. While formal guidance may be limited in Asia-Pacific, principles from leading agencies (FDA, EMA) are being adopted regionally. AI tools are recognized as helpful in navigating complex regulatory landscapes and harmonizing information.

Harmonization and Mutual Recognition of Approvals

Currently, Japan, Korea, and Singapore operate largely independent regulatory systems without formal mutual recognition of approvals. Singapore offers an accelerated approval route for products approved by five reference agencies (Australia TGA, Health Canada, US FDA, EMA, UK MHRA). Harmonization efforts are ongoing but face challenges due to differences in national health insurance coverage, localized industry presence, and regulatory specifics. Panelists expressed optimism about future collaboration and harmonization to benefit regional development and patient access.

Recommendations for Sponsors Seeking Approval in Asia-Pacific

Engage early and proactively with local regulators to clarify requirements and expectations. Reference the ISCT APAC Industry Committee’s regulatory roadmap and guidance papers for detailed regional insights.

Utilize AI tools for regulatory comparison and translation but verify outputs carefully. Understand and prepare for dual regulatory pathways (traditional pharmaceutical and innovative access routes). Foster inclusion of local clinical trial sites to facilitate bridging studies and regulatory acceptance.

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The paper is available on Cytotherapy: https://www.isct-cytotherapy.org/article/S1465-3249(25)00590-0/fulltext

If you are interested in knowing more about our initiatives, please visit: Asia Pacific Industry Committee: https://www.isctglobal.org/about/isct-committees/apac-industry