EMA outlines tailored clinical approach for the development of biosimilars

“Biosimilars may be approved without providing CES (Comparative Clinical Efficacy Studies) or even PD data (Pharmacodynamic) if similar clinical efficacy and safety pharmacology can be inferred from a sufficiently stringent evaluation of analytical comparability, in vitro pharmacology, and a comparative clinical PK (Pharmacokinetic) trial”, is the conclusion of the “Reflection paper on a tailored clinical approach in biosimilar development” the EMA has just released for public consultation.

We at Formycon clearly welcome this approach, as it has the potential to accelerate biosimilar development and reduce costs significantly. In the long term, this could also reduce the often-described biosimilar void (the number of off-patent biologics without biosimilar competition) and improve patient access to biologic therapies. With regard to our decision to discontinue the Phase III trial of our biosimilar candidate FYB206 we see the reflection paper as confirmation. We believe that the EMA’s considerations support our approach of demonstrating the therapeutic comparability of FYB206 with the reference drug Keytruda® based on comprehensive analytical data and the data from the Phase I study (Dahlia PK study).

According to the EMA draft, in terms of clinical development, PK studies (Phase-I-Studies) are in general sufficient for the approval of a biosimilar. CES studies (Phase-III-Studies) may still be required in cases where a “biological is not well-characterisable and/or has an unknown or poorly understood Mode of Action, structure-function relationship, or if the impact of observed differences on clinical outcomes is unclear”. To address any remaining uncertainty regarding comparability in exposure, safety and immunogenicity, the comparative PK study will be adapted in the reflected tailored clinical approach.

The published draft can be reviewed and commented on during the public consultation period until the end of September 2025: https://www.ema.europa.eu/en/documents/other/reflection-paper-tailored-clinical-approach-biosimilar-development_en.pdf