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Summary

Biologics and Advanced Therapy Medicinal Products (ATMPs) require potency assays for release. Immature assays can delay regulatory approval, as seen with Iovance and Mesoblast, but issues are being resolved.

Keywords:
ATMPMAAblacommercialisationfunctional assayquality control

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Area of Science:

  • Biotechnology
  • Pharmaceutical Sciences
  • Regulatory Affairs

Background:

  • Biologics and Advanced Therapy Medicinal Products (ATMPs) have unique release criteria, including a "potency" standard, unlike conventional pharmaceuticals.
  • Regulatory agencies often permit the use of early-stage potency assays during clinical trials, with the expectation of future validation.

Purpose of the Study:

  • To highlight the challenges and implications of using continuously developed potency assays for biologics and ATMPs.
  • To examine the impact of immature potency assays on regulatory submissions, using specific company examples.

Main Methods:

  • Review of regulatory expectations for potency assays in early-phase clinical trials.
  • Analysis of case studies (Iovance, Mesoblast) involving potency assay development and regulatory submissions.
  • Examination of recent regulatory decisions regarding Biologics License Application (BLA) and Marketing Authorisation Application (MAA) for ATMPs.

Main Results:

  • The continuous development model for potency assays has led to regulatory rejections for companies like Iovance and Mesoblast despite successful Phase III trials.
  • Recent regulatory acceptances for Mesoblast's Remestemcel and Iovance's Lifileucel indicate that issues with potency assays can be overcome.

Conclusions:

  • The development and validation of robust potency assays are critical for the successful regulatory approval of biologics and ATMPs.
  • Addressing potency assay deficiencies proactively throughout clinical development is essential to avoid submission delays and rejections.