Regulatory
Rapporteur

 

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May 2025  |  Vol. 22  |  No. 5

EDITORIAL

Advanced therapies: A maturing regulatory framework

The May issue of Regulatory Rapporteur focuses on advanced therapy medicinal products (ATMPs).

With growing numbers of ATMPs being approved in both the EU and the US, and a large and diverse pipeline of ATMPs in late-stage development, from a regulatory perspective, the framework has reached a certain level of maturity. However, with new innovations continually emerging, industry and regulators need to work together to ensure that frameworks remain fit for purpose. In this issue, we explore recent transformative ATMP developments and the opportunities and challenges that these pose.

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By Marie Uguen

FOCUS

Gene Therapy Medicinal Products: An examination of regulatory CMC requirements

Gene Therapy Medicinal Products (GTMPs) represent a transformative medical innovation with the potential to cure previously untreatable genetic disorders. However, the journey from clinical development to commercial production is complex, particularly when it comes to meeting stringent regulatory chemistry, manufacturing and controls (CMC) requirements. This article explores the CMC landscape for gene therapies, emphasising the critical elements necessary to ensure quality, safety and efficacy throughout the product lifecycle.

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By Srinivas Yerraguntla, Achuta Raju Samala, Rajendra Kunda, Indira Priyadarsini, Asha Balakrishnan, Sateesh Babu GSV, Deepti Jagga, Hao Liu, Vivek Tomar and Allison Gillespie, all of Syneos Health

FOCUS

Recent advances and the near-term outlook for Advanced Therapy Medicinal Products in cancer treatment

The field of oncology has seen significant medicinal advances in recent years, with ATMPs bringing new promise to the treatment of cancers. This article assesses some of the most recent notable clinical developments, where the next generation of research is heading, how regulatory authorities are responding to these advances, and how these developments and regulatory proposals are likely to impact patient access.

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By Jörg Schneider, Alexia Blesius and Christian K Schneider, all of PharmaLex

FOCUS

Case studies: The impact of the In Vitro Diagnostic Regulation on ATMP clinical trials and drug development plans

The implementation of the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 (IVDR) has significantly impacted the initiation of clinical trials (CTs) with ATMPs in Europe. The challenges faced are illustrated through three case studies of CTs involving adeno-associated virus (AAV) vector-based gene therapy for rare diseases, and a list of key IVD-related questions are provided to help identify future CT requirements.

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By Annelise Brossel, Hélène Lafolly, Valérie Salentey and Damien Stalin, on behalf of the France Biotech ATMP Commission

STANDALONE

Navigating the FDA’s platform technology designation: Key insights and best practices for drug manufacturers

Platform technologies can be crucial in transforming the development of advanced drugs and biologics by optimising manufacturing processes while improving efficiency and quality. This article discusses the US Food and Drug Administration’s (FDA) Platform Technology Designation initiative, including its key elements, challenges, and its significance in the regulatory landscape.

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By Sree Vidya Manur and Likhitha Mateti

STANDALONE

Navigating orphan drug designation in the EU

Orphan drug designation (ODD) in the EU is a regulatory mechanism designed to encourage the development of treatments for rare diseases, which often lack sufficient therapeutic options. This article explores the legal framework of ODD, the eligibility criteria and the key considerations for pharmaceutical companies seeking this designation. It also identifies some of the challenges associated with demonstrating significant benefits, obtaining reliable prevalence data and aligning with evolving regulatory requirements.

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By Francisco Baptista of Arriello Ireland Limited

STANDALONE

Prescribing efficiency: Using generative artificial intelligence for pharmacovigilance

The potential of generative artificial intelligence (AI) in streamlining the processes involved in pharmacovigilance has been tested and the results are promising. This article aims to explore how generative AI can optimise pharmacovigilance workflows, reduce reporting burdens and improve accuracy and timeliness across the pharmaceutical lifecycle.

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By Ihab Mansoor and Matthew Rector of Narrativa