Fajar Dumadi, BSc
Specialist, Regulatory Program
ISCT
Canada
With great excitement, the International Society for Cell & Gene Therapy (ISCT) publishes its fifth Global Regulatory Report in collaboration with Clarivate™. This report offers a comprehensive global overview of the cell and gene therapy regulatory landscape, including pipeline, late-stage (Phase III and pre-registration), and approved products (Cell, Genetically-Modified Cell, and Gene Therapies).
Highlights from this issue include:
- First Approval
- BBM H901, the first gene therapy for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency), received its approval from National Medical Products Administration (NMPA).
- Encelto™, the first cell-based gene therapy for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel), received its approval from US Food and Drug Administration (US FDA).
- Papzimeos™, the first-of-its-kind non-replicating adenoviral vector-based immunotherapy for the treatment of adult patients with recurrent respiratory papillomatosis (RRP), received its approval from US Food and Drug Administration (US FDA).
- Pulidekai®, the first approved CAR-T therapy for relapsed or refractory B-cell acute lymphoblastic leukemia in pediatric patients, received its approval from National Medical Products Administration (NMPA).
- Qartemi®, the first approved CAR-T therapy for relapsed or refractory B-cell Non-Hodgkin Lymphoma (B-NHL), received its approval from Central Drugs Standard Control Organization (CDSCO).
- Ruibosheng, the first human umbilical cord-derived mesenchymal stem cell therapy for graft-versus-host disease, received its conditional approval from National Medical Products Administration (NMPA).
- Waskyra™, the first gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS), received its approval from US Food and Drug Administration (US FDA).
- Zevaskyn®, the first autologous cell-based gene therapy for for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), received its approval from US Food and Drug Administration (US FDA).
- Key Global Legislative/Framework Changes
- EMA - Guideline on quality, non-clinical and clinical requirements for investigational ATMPs in clinical trials.
- Health Canada - A framework for regulating and authorizing advanced therapeutic products.
- HSA - Guidance on cell, tissue and gene therapy products registration and variation applications in Singapore.
- ICH - Guideline on Good Clinical Practice (E6(R3)).
- MHRA - Human Medicines Modular Manufacture and Point of Care Regulations 2025.
- State Council of the People's Republic of China - Regulations on the clinical research and clinical translational application of biomedical new technologies (Order No. 818).
- US FDA - Draft Guidance on post-approval methods to capture safety and efficacy data for cell and gene therapy products.
Additionally, as we look back to 2025:
- Early‑stage development continues to drive innovation, with Phase I and Phase II studies accounting for the majority of the clinical trial pipeline worldwide.
- Genetically-modified cell therapies continue to lead the market, representing 46% of approved products and 53% of all clinical trials.
- Gene therapies demonstrate strong momentum, accounting for 41% of pre‑registration candidates and ranking as the second‑largest category in clinical trials at 25%.
Bambi Grilley, ISCT Chief Regulatory Officer, remarked, "I am so excited to share the latest edition of the ISCT Global Regulatory Report. With early‑stage trials driving the majority of the global pipeline, and genetically modified cell therapies and gene therapies continuing to lead both approvals and pre‑registration activity, regulatory strategy is increasingly shaping how innovation translates into patient access."
Join us in celebrating the release of this new edition. [Click here to access the full report]
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