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Regulatory Rapporteur

 

May 2025  |  Volume 22  |  No. 5

 

 

While both industry and payers are still looking for a workable pricing and reimbursement system for advanced therapy medicinal products (ATMPs), from a regulatory professional’s viewpoint, the ATMP regulatory framework (encompassing regulations, guidelines, pathways, incentives and Q&As) has reached a certain level of maturity. This is evidenced by the number of ATMPs approved over the past decade: 19 are currently authorised in the EU with 9 new ATMPs currently under evaluation,[1] 44 cellular and gene therapy (CGT) products are so far approved in the US,[2] and there is a further large and diverse pipeline of ATMPs in late-stage development.[3]

In this annual Regulatory Rapporteur issue dedicated to ATMPs, we deep dive into chemistry, manufacturing and controls (CMC) requirements and how they evolve throughout product development, we look at the most recent therapeutic innovations in the oncology field, and discuss the complex interconnection between ATMPs and in vitro diagnostic medical devices. All these articles have a common motto: it’s never too early to engage with regulators!

 

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Our first article, by Srinivas Yerraguntla, Achuta Raju Samala, Rajendra Kunda, Indira Priyadarsini, Asha Balakrishnan, Sateesh Babu, Deepti Jagga, Hao Liu, Vivek Tomar and Allison Gillespie at Syneos Health, tackles the progression of regulatory CMC requirements for gene therapy medicinal products from early clinical development phases up to registration and beyond. The article provides an in-depth exploration of the landscape, and describes the unique challenges of scalability, purity and potency, stability and comparability, as well as the importance of robust control strategies for critical quality attributes.

Our second article, written by Jörg Schneider, Alexia Blesius and Christian K Schneider at PharmaLex, assesses some of the most recent and notable ATMP clinical developments in the oncology field, where significant medicinal advances have been observed. The article covers CAR-T cell therapies, which have played a transformational role in cancer treatment (particularly for blood cancers), oncolytic virus treatment modalities and cancer vaccines. The authors highlight the increasingly key role of regulators in enabling innovation with the setting up of dedicated regulatory frameworks, while maintaining their safety ‘gatekeeper’ mandate.

In our third article, Annelise Brossel, Hélène Lafolly, Valérie Salentey and Damien Stalin from the France Biotech ATMP Commission have analysed the impact of the In Vitro Diagnostic Medical Devices Regulation (IVDR) on ATMP clinical trials and the overall drug development plan. They highlight how the implementation of the IVDR, which came into application in mid-2022, has significantly impacted the initiation of clinical trials with ATMPs in Europe. They illustrate the new challenges through three case studies focused on AAV-vector-based gene therapy, and share a very useful list of key IVD-related questions to be considered by sponsors.

In May 2024, the FDA issued a foundational draft guidance for sponsors manufacturing drugs under platform technology modalities, introducing the Platform Technology Designation (PTD). While not specific to ATMPs, these complex biologics definitely fall under the scope of the new guidance. Sree Vidya Manur and Likhitha Mateti delve into the PTD initiative, its key elements, challenges and its significance in the regulatory landscape. They also explore the activities and strategies for sponsors at various stages, from pre-approval preparation to post-approval management.

Moving to other areas of interest, in our fifth article, Francisco Baptista of Arriello Ireland Limited explores the legal framework of orphan drug designation, the eligibility criteria and the key considerations for sponsors, with a focus on the European Union. This booster shot article also identifies some of the challenges associated with demonstrating significant benefit, obtaining reliable prevalence data and aligning with evolving regulatory requirements.

In our final article, Ihab Mansoor and Matthew Rector of Narrativa look at the potential of generative artificial intelligence (GenAI) in streamlining pharmacovigilance processes and discuss some of its possible limitations.

I hope you enjoy reading these articles as much as I did.

 

 

References

[1] European Medicines Agency (EMA) ‘Medicines for human use under evaluation’. (Accessed: 1 May 2025).

[2] US Food and Drug Administration (FDA) ‘Approved Cellular and Gene Therapy Products’. (Accessed: 1 May 2025).

[3] Add Alliance for Regenerative Medicine (2024) ‘Ongoing Clinical Trials by Phase and Therapeutic Approach’. (Accessed: 1 May 2025).

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