With great excitement, the International Society for Cell & Therapy (ISCT) publishes its fourth bi-annual Global Regulatory Report in collaboration with Citeline. Similar to the previous editions, 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:
- RegeneCyte™, a vital cord blood stem cell therapy for transplantation in patients with blood and immune system disorders, has received approval from US FDA
- Akuugo®, a human somatic stem cell product indicated for improving chronic motor paralysis resulting from traumatic brain injury, has received approval from Japan PMDA
- Tecelra®, the first gene therapy to treat adults with metastatic synovial sarcoma, has received approval from US FDA
- Aucatzyl®, cell therapy to treat adults with relapsed or refractory B-cell precursor Acute Lymphoblastic Leukemia (ALL), has received its approval from US FDA
- Ryoncil®, the first FDA-approved MSC therapy indicated for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients 2 months of age and older
- Key global legislative/framework changes
- Joint scientific consultation on EU Health Technology Assessment
- Public consultation on proposed amendments to the Australian Gene Technology Act 2000 (‘GT Act’)—the first major changes made to the Act since it was first implemented over 20 years ago
- Public consultation on US FDA draft guidance on Expedited Program for Serious Conditions – Accelerated Approval of Drugs and Biologics
H2 2024 marked a period of growth for both genetically and non-genetically modified cell therapies, as well as gene therapies:
- The number of genetically and non-genetically modified cell therapies in the pipeline has grown by over 5% since H1 2024
- The non-genetically modified cell category continues to dominate the pipeline, with 45 therapies in phase III/Pre-Registration phase and 67% of all approved products
- The number of gene therapies in the pre-registration phase has increased significantly, five times higher than in H1 2024
Bambi Grilley, ISCT Chief Regulatory Officer, remarked, "I am excited to share the latest edition of the ISCT Global Regulatory Report! While non-modified cell therapies continue to dominate the pipeline, other categories are also on the rise. Genetically modified therapies saw a 5% growth, and a significant number of gene therapies have now entered the pre-registration phase—marking a positive shift in the field".
Join us in celebrating the release of this new edition.
[Click here to access the full report]
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