Cell and Gene Therapy Products: Regulatory Strategy Across the US and EU

Cell and gene therapies are redefining the therapeutic landscape with the potential to treat diseases ranging from rare genetic disorders to cancers. However, their complexity brings unique challenges, especially in navigating the regulatory pathways required for clinical development and commercialization. Understanding the distinct ways in which these products receive approval in the European Union and United States is essential to accelerate development and eventual patient access for these therapies.

In the US, cell and gene therapies are regulated as biologics and are reviewed primarily by the Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER). Commercialization of these therapies requires a biologics license application (BLA) for approval. In Europe, cell and gene therapies fall under a distinct regulatory class, Advanced Therapy Medicinal Products (ATMPs), which include four categories (gene therapy products, somatic cell therapy products, tissue-engineered products, and combined ATMPs). These products are reviewed by the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) and require a Marketing Authorization Application (MAA) for approval.

While many therapies approved by the FDA also hold market authorization from the EMA and vice versa, not all do. Certain products approved by the FDA in the US have faced obstacles in Europe. For example, Elevidys, developed by Sarepta Therapeutics to treat Duchenne Muscular Dystrophy, has encountered regulatory challenges with the EMA due to concerns about whether the clinical data sufficiently demonstrates a favorable benefit-risk profile. Similarly, some therapies authorized in the EU have not secured FDA approval. Ebvallo, which is approved in the EU, has failed to gain US approval due to factors such as third-party manufacturing and study design issues. These examples underscore how differing standards, regulatory priorities, and benefit-risk assessments between agencies can significantly influence the global availability of cell and gene therapies. The table below showcases the approved cell and gene therapy products in the US and in the EU.

Trends & Regulatory Advances

When manufacturers are developing cell and gene therapies, several factors need to be kept in mind:

• Regulatory Flexibility: A one-size-fits-all regulatory strategy is not feasible as the US FDA and EU EMA operate under different regulatory frameworks, each with distinct requirements for study design, marketing authorization procedures, and post-approval oversight.
• Approval Timelines: Historically, the FDA has often approved therapies before the EMA, with varying evidentiary expectations, dataset requirements, and review processes.
• Evolving Landscape: The regulatory environment for cell and gene therapies is continuously evolving, requiring ongoing strategic adaptation to ensure products remain compliant and to minimize the risk of delays or potential failure.