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EMA Adopts Guidance on Medical Products Containing Genetically Modified Cells

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By Alexander Gaffney

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The European Medicines Agency (EMA) announced 5 May it has adopted a scientific guideline on aspects relating to the development and testing of medicinal products containing genetically modified cells.

The scientific guideline addresses the three main areas of drug development: quality considerations within the manufacturing process, non-clinical testing and clinical testing.

EMA notes genetically modified cells may either be used as therapies themselves or as part of the manufacturing process of an engineered product, but notes the same Guideline on human cell-based medicinal products (EMEA/CHMP/410869/2006) should be followed, along with any other applicable guidelines.

EMA further notes that a risk-based approach should be taken when evaluating genetically modified cell products, as each individual cell’s level of risk is “highly dependent on the origin of the cell, the type of vector and the method of gene transfer used for the genetic modification, the manufacturing process, the non-cellular components and the specific therapeutic use” of a product.

Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells describes the considerations that must be addressed when manufacturing and testing each genetically modified cell product, including:

  • Quality considerations: Starting materials, other materials, reagents, excipients, the manufacturing process, cell preparation and culture, gene transfer, further manufacturing steps, in-process controls, process validation, characterization, identity testing, purity testing, potency testing, release criteria and stability testing.
  • Non-Clinical testing considerations: Pharmacodynamic testing, pharmacokinetic testing and toxicology testing.
  • Clinical testing considerations: Clinical trial design, dose selection, pharmacodynamic testing, immunogenicity testing, efficacy, safety and follow-up testing.

EMA also notes some of the considerations necessary for pharmacovigilance, including the possibility for long-term safety issues including infections, immunogenicity, immunosuppression, malignant transformation and the “durability of the associated medical device/biomaterial component.” All such factors must be addressed in a Risk Management Plan.


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EMA - Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells




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