Breakthroughs in biotechnology are transforming how we approach some of the most challenging medical conditions. Genetic disorders, late-stage cancers, and severe tissue damage are increasingly being treated with therapies tailored to each individual’s unique biology. From gene therapies that rewrite faulty DNA to cell-based treatments that regenerate damaged tissue, advanced therapies are not just reshaping medicine they’re redefining what’s possible.
As we move through 2025, the field is experiencing remarkable growth, driven by scientific breakthroughs, increased investment, and evolving regulatory frameworks.
What Are Advanced Therapies?
Advanced therapies, comprising gene therapy, cell therapy, and tissue engineering, are treatments developed to address serious diseases, often where no effective therapies currently exist. These innovative approaches work by:
- Modifying or replacing faulty genes
- Harnessing living cells to fight disease
- Regenerating or repairing damaged tissues
Unlike traditional small-molecule drugs, these “living medicines” contain genetic material or viable cells, often enabling single-dose, potentially curative effects. Their targeted nature offers a new paradigm in treating complex conditions such as cancer, genetic disorders, and rare diseases.
Market Outlook: Accelerated Growth Ahead
The market for Advanced Therapy Medicinal Products (ATMPs) is on a steep upward trajectory, reflecting both increasing investment and growing demand for innovative treatments. According to recent industry reports:
- The global ATMP market was valued at USD 14.67 billion in 2024, and is projected to reach USD 39.55 billion by 2032, growing at a compound annual growth rate (CAGR) of 13.2%.
- Another forecast suggests the market could reach USD 170.47 billion by 2034, with a CAGR of 16.83% from 2025 to 2034.
North America, particularly the United States, currently holds the largest market share, driven by a high prevalence of cancer and neurological disorders, as well as significant private sector investment. However, Europe and the Asia-Pacific region are also accelerating progress through regulatory reforms and increased public-private collaboration.
Collaborating for Global Success in Advanced Therapy Medicinal Products (ATMPs)
Regulatory Landscape: Evolving with Innovation
As advanced therapies continue to evolve, global regulatory frameworks are adapting to keep pace with innovation. Regulatory bodies across key markets are establishing specialized pathways and designations to expedite development and ensure patient safety.
European Union: EMA’s ATMP Framework
In the European Union (EU), the European Medicines Agency (EMA) regulates ATMPs under Regulation (EC) No 1394/2007, which includes gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products.
The Committee for Advanced Therapies (CAT) plays a central role in assessing ATMPs, evaluating quality, safety, and efficacy. The CAT prepares a draft opinion that is submitted to the Committee for Medicinal Products for Human Use (CHMP) for final recommendations. The European Commission subsequently grants centralized marketing authorization, typically within about seven months (with CHMP opinion issued by Day 210 and Commission decision by Day 277).
To further support innovation, the EMA offers expedited development pathways such as PRIority Medicines (PRIME) or PRIME Designation, which accelerates review for therapies addressing unmet medical needs. Notably, in September 2022, the EMA launched a pilot program for academic and non-profit developers of ATMPs, providing scientific guidance, fee reductions, and waivers to promote development of treatments for rare diseases.
United States: FDA’s RMAT Designation
In the United States, the Food and Drug Administration (FDA) regulates advanced therapies through the Center for Biologics Evaluation and Research (CBER) and its Office of Tissues and Advanced Therapies (OTAT). A key initiative is the Regenerative Medicine Advanced Therapy (RMAT) designation, which aims to expedite the development and review of promising regenerative therapies.
To qualify for RMAT, a therapy must:
- Be a regenerative medicine product (such as a gene therapy, cell therapy, or tissue-engineered product)
- Target a serious or life-threatening condition
- Provide preliminary clinical evidence demonstrating the potential to address unmet medical needs
Developers must submit an Investigational New Drug (IND) application to begin clinical trials and a Biologics License Application (BLA) for marketing authorization. The FDA also provides access to additional expedited programs, including Breakthrough Therapy Designation, Fast Track, and Accelerated Approval.
As of 2022, the FDA had received 187 RMAT designation requests, with 72 granted indicating strong interest and the selective nature of the program.
Japan: PMDA’s Conditional Approval System
Japan has established one of the most progressive regulatory frameworks for regenerative medicine. The Pharmaceuticals and Medical Devices Agency (PMDA) oversees ATMPs under the Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act (PMD Act) and the Act on the Safety of Regenerative Medicine (ASRM), both enacted in 2014.
To promote early access to novel therapies, Japan offers:
- A conditional and time-limited approval system, allowing market entry based on promising early-phase clinical data (typically Phase II), with a requirement to conduct post-marketing studies.
- The Sakigake (Pioneer) designation, which accelerates development and regulatory review for first-in-class, innovative therapies expected to be submitted in Japan before any other country.
These frameworks are designed to balance timely access to cutting-edge treatments with ongoing safety evaluation.
Emerging Markets & Global Harmonization
While the EU, U.S., and Japan lead in ATMP regulation, other global regions are developing or enhancing their regulatory capabilities through international collaboration and harmonization efforts.
The World Health Organization (WHO) plays a key role in promoting regulatory convergence by advocating for:
- Regulatory reliance, enabling countries to use assessments conducted by trusted regulatory authorities
- Joint reviews and shared inspections, particularly for advanced therapies and Human Cells and Tissue (HCTs)
Programs such as the African Medicines Regulatory Harmonisation (AMRH) are helping African regulators collaborate with more established agencies like the EMA to build local capacity. Some countries are also piloting the use of EMA scientific assessments to support national regulatory decisions.
Additionally, global initiatives like the Pharmaceutical Inspection Co-operation Scheme (PIC/S) work with regulatory bodies including Zimbabwe’s Medicines Control Authority to strengthen inspection readiness and promote consistent quality and safety standards.
Conclusion
Advanced therapies are ushering in a new era of medicine, offering hope to patients with diseases previously considered untreatable. In 2025, the field is characterized by rapid growth, active regulatory innovation, and a shared commitment to addressing clinical unmet needs.
As regulatory agencies adapt to scientific progress, they are implementing guidelines, launching pilot programs, and taking action against unregulated therapies to ensure both safety and access. Yet challenges remain, particularly around manufacturing complexity, cost of goods, and market access.
Realizing the full potential of advanced therapies will require collaboration between regulators, developers, payers, and global health organizations to ensure these therapies reach the patients who need them most.
How DDReg Can Help
DDReg offers comprehensive Life sciences regulatory support across the entire product lifecycle for advanced therapies both globally and within key local markets. Our multidisciplinary team provides up-to-date guidance aligned with evolving regional requirements, helping you navigate complex regulatory landscapes with confidence.
Whether you’re launching a novel ATMP, addressing post-market challenges, or expanding to new markets, DDReg delivers the insight and expertise needed to ensure success.
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