Core36: Advanced therapy medicinal products. Vaccines

🧫 High-yield / 要点：Advanced therapy medicinal products ATMPs are gene-, cell-, and tissue-based medicines. They differ from small molecules and biologics because they contain active cells or genetic constructs.

Definition / 定義

• ATMPs = innovative medicines based on:
  • Gene therapy.
  • Cell therapy.
  • Tissue therapy.
• They may act by:
  • Metabolic mechanisms.
  • Immunological mechanisms.
  • Genetic mechanisms.
  • Other non-classical pharmacological mechanisms.

Key features / 特徴

• Technically demanding to develop and manufacture.
• Current market success is limited.
• The Committee for Advanced Therapies / CAT has a central role in scientific evaluation.

EMA categories / EMA分類

Category	Meaning	Example
GTMP	Gene therapy medicinal product: therapeutic genes introduced by recombinant technology	Imlygic for unresectable melanoma
SCTMP	Somatic cell therapy medicinal product: cells/tissues with changed biological characteristics	Provenge for metastatic castration-resistant prostate cancer
TEP	Tissue engineered product: modified cells/tissues for repair, regeneration, replacement	Holoclar for limbal stem cell deficiency after eye burns
Combined ATMP	Integrated product combining multiple methods	Cells embedded in biodegradable matrix

Remember / 覚え方

• ATMP = gene/cell/tissue therapy
• More complex than small molecules and ordinary biologics
• CAT evaluates ATMPs scientifically