NorthWest Biotherapeutics Inc (NWBO)

[Lykiri]

The following is from a letter from the European Commission- Advanced Therapies:

The concept of similarity in an ATMP setting.

This Q&A document addresses questions that have been raised by developers of ATMPs regarding the application of the concept of “similar active substance” in an ATMP setting. The Q&A document will be updated in light of accumulated experience during the assessment of these medicinal products.

https://ec.europa.eu/health/human-use/advanced-therapies_en

QUESTIONS AND ANSWERS RELATED TO THE ASSESSMENT OF SIMILARITY FOR ADVANCED THERAPY MEDICINAL PRODUCTS ("ATMPS") IN THE CONTEXT OF THE ORPHAN LEGISLATION.
FREQUENTLY ASKED QUESTIONS
VERSION 1
Date: 25 May 2018

In the case of ATMPs, differences in the manufacturing technology can be relevant to demonstrate non-similarity between two products. What is the meaning of "manufacturing technology"? Certain technological changes to the manufacturing process of ATMPs can justify a finding of non-similarity. The following examples (non-exhaustive list) illustrate differences in manufacturing technologies that could support a claim of non-similarity, provided that those changes lead to a significant impact on the biological characteristics and/or biological activity which is relevant for the intended therapeutic effect and/or safety attributes of the product:

? CD34+ cells transduced with a viral vector could be considered to be non-similar to CD34+ cells transduced with gene editing technology, even if both products target the same indication.

? Having regard to the different precision, efficiency, and specificity profiles of the different meganuclease-based engineering technologies, a gene therapy medicinal product developed with zinc-finger nucleases and a gene therapy medicinal product developed with CRISPR-Cas9 could be considered to be non-similar, even if they target the same DNA sequence.

? Dendritic cells activated with a tumour lysate could be considered to be nonsimilar to dendritic cells activated by means of purified tumour protein, even if both products target the same indication.


It is stressed that not all changes to the manufacturing process can qualify as a change in manufacturing technology. For example, a change in manufacturing process (e.g. cultivation of cells in an open system vs. a closed system; change in the number of cell passages) or a change in equipment (e.g. upgraded bioreactor) generally cannot support a finding of non-similarity.

https://ec.europa.eu/health/sites/health/files/files/orphanmp/doc/2018_qa_atmps_en.pdf

Someone any thought???