AIHTA - Publications - Search - CAR-T Cell Therapies, ATMP and Gene Therapies in Development, Horizon Scanning: ATMPs and Gene Therapies

Wild, C. and Wohlhöfner, K. and Sehic, O. and Grössmann, N. (2020): CAR-T Cell Therapies, ATMP and Gene Therapies in Development, Horizon Scanning: ATMPs and Gene Therapies. AIHTA Policy Brief 006b.

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It is predicted that by 2022 there will be about 16 "Advanced Therapies Medicinal Products" (ATMPs) on the market, about 2-4 new therapies per year. There are currently 11 ATMPs approved. According to the annual report of the "Alliance of Regenerative Medicine (ARM)", by the end of 2019 more than 1,000 clinical trials worldwide were investigating regenerative/ advanced therapies for various diseases, 94 of which are in phase 3, 591 in phase 2, 60% in oncological indications alone. Among the best known already approved are the CAR-T cell therapies (2 approved in Europe, 1 in the approval process) and gene therapies for spinal muscular atrophy (also 2 approved in Europe, 1 in the approval process). Two Horizon Scanning reports –a cooperation project of AIHTA and Tirol Kliniken- have dealt with a look into the near future of further gene therapies.

The European Medicines Agency (EMA) classifies ATMPs into three main types: Gene therapeutics and somatic cell therapeutic drugs and tissue engineering products. The two reports should address the questions for which indications CAR-T cell therapies and other ATMPs/gene therapies are in development and by when a marketing authorisation is expected. To answer the questions, a systematic search of study registries was performed to identify gene therapies in development and ATMPs, followed by the extraction of data on the identified ongoing clinical trials, complemented by a search of the EMA database of drugs under evaluation to identify those therapies that are expected to be approved soon. Finally, published information on the products, their indication and patient population was collected. The search identified 32 ATMPs in 8 broad indication areas and 13 CAR-T cell therapies in haematological malignancies at an advanced stage of development.

Item Type:AIHTA Policy Brief
Keywords:Horizon Scanning, ATMP, gene therapy, CAR-T cell therapy, rare diseases
Subjects:QT Physiology > QT 34-37 Physics. Mathematics. Engineering > QT 36-37 Biomedical engineering, nanotechnology, biocompatible materials
QU Biochemistry > QU 300-375 Cells
QU Biochemistry > QU 450-500 Genetics
QV Pharmacology, toxicology, pharmacy > QV 38 Pharmacogenetics
QW Microbiology. Immunology > QW 501-949 Immunology
QZ Pathology > QZ 50 Medical genetics
QZ Pathology > QZ 200-380 Neoplasms.Cysts
W Health professions > W 84 Health services. Quality of health care
WB Practice of medicine > WB 300-962 Therapeutics
WB Practice of medicine > WB 340-356 Drug Administration
Series Name:AIHTA Policy Brief 006b
Deposited on:26 Aug 2020 10:09
Last Modified:15 Apr 2021 14:18

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