ATMP | Pharmaceuticals | Regulatory Rapporteur

Advanced Therapy Medicinal Products

PS2: ATMP innovations and clinical trials with genetically modified organisms
2023-12-05T16:00:00+00:00By Davina Stevenson
Session leader:
Evolution of GMO requirements for innovative investigational medicinal products upon transition to the EU CTR
2023-09-14T09:27:00+01:00By Sabine Ruehle, Kathryn Parsley, Gabriel Bohl, Myra Widjojoatmodjo, Janneke Westra-de Vlieger, Jayne Hunt,
supportive members of the TOPRA Biotech and Clinical Trial SPIN groups, Pierre-Frederic Omnes
The EU CTR replaced the EU CTD on 31 January 2022, which impacts investigational medicinal products that fall under genetically modified organisms (GMO-IMPs).
An overview of the regulatory framework for advanced therapies
2023-09-14T09:14:00+01:00By Regulatory Affairs Workgroup SINDUSFARMA
Advanced therapy medicinal products (ATMPs) have had recent advances in development and have already been subject to regulation in some markets. This article summarises the regulatory framework for ATMPs in some of the markets where these requirements are in place: Argentina, Brazil, Europe, and the US. The categories analysed were regulatory classification, product categories, packaging, product insert, good manufacturing practice (GMP), pharmacovigilance, environmental safety evaluation, clinical trial application (CTA) and investigational new drug (IND) application, post-approval changes, chemistry, manufacturing and controls (CMC)/quality, importation process, prioritisation pathways, transport validation, local release testing and traceability.
FDA's Emerging Technology Programme
2023-07-18T09:42:00+01:00By Catrin Jackson
The FDA’s Emerging Technology Programme intends to assist industry in gaining regulatory approval for innovative technology drug applications.
RegRapPod – May 2023
2023-05-31T17:00:00+01:00
Vol. 20 #5
In this episode of RegRapPod, host Alan Booth, the managing editor of Regulatory Rapporteur, hears from May’s Issue Editor, Harriet Edwards, about the rapid developments in advanced therapy medicinal products and the tensions that arise between regulation and innovation.
Regulating Vaxzevria
2023-05-26T11:00:00+01:00
Vol. 20 #4
MEMBER EXCLUSIVE:
Hosted by Alan Booth, managing editor of Regulatory Rapporteur, this episode of RegRapPod is an extended conversation with authors Priti Shah and Anna Litsiou following on from their article in the April issue [Vol. 20 #4]. Priti and Anna share insights around the regulatory process of AstraZeneca’s COVID-19 vaccine - Vaxzevria.
Gene therapy gives hope to patients with ‘butterfly skin’
2023-05-25T11:03:00+01:00By Catrin Jackson
FDA approves first gene therapy to treat dystrophic epidermolysis
bullosa patients – a condition sometimes called ‘butterfly skin’.
Evolution of GMO requirements for innovative investigational medicinal products upon transition to the EU CTR
2023-05-18T12:58:00+01:00By Sabine Ruehle, Pierre Omnes, Kathryn Parsley, Gabriel Bohl, Myra Widjojoatmodjo, Janneke Westra-de Vlieger, Jayne Hunt,
supportive members of the TOPRA Biotech and Clinical Trial SPIN groups
The EU CTR replaced the EU CTD on 31 January 2022, which impacts investigational medicinal products that fall under genetically modified organisms (GMO-IMPs).
G-E-T-ting there – how gene editing technologies could change more than genes
2023-05-18T12:57:00+01:00By Harriet Edwards
The field of genome editing technologies (GETs) is rapidly advancing, marking a new and exciting era of curative medicines. These products can alter genetic material in patients, highlighting the crucial need to introduce dedicated legislation to ensure that GETs are developed and maintained in a safe and effective manner.
Alternatives to animal testing
in the development of ATMPs
2023-05-18T12:56:00+01:00By Michaela Sharpe
This article looks at a range of innovative non-animal test methods, including in vitro tests based on human and animal cells, genomic technologies and in silico modelling.
An overview of the regulatory framework for advanced therapies
2023-04-06T07:57:00+01:00By Regulatory Affairs Workgroup SINDUSFARMA
Advanced therapy medicinal products (ATMPs) have recently advanced in development and are subject to regulation in some markets. This article summarises the regulatory framework for ATMPs in some of the markets where these requirements are in place: Argentina, Brazil, Europe, and the US.
HM8: The present and future of developing and commercialising cell and gene therapies in a global world
2022-12-05T00:14:00+00:00By Nathalie Bourrit
Gene therapies are highly innovative products which have been approved only for the treatment of rare diseases so far. Changes in manufacturing processes during development as well as difficulties in gathering large clinical data sets explain the common challenges encountered by gene therapy developers to demonstrate the benefit of their ATMP.
FDA meetings: How to hold a successful meeting across the drug development lifecycle
2022-06-24T16:13:00+01:00By Daniela Drago, Danielle Helton Corwin
This continuing professional development supplement discusses the available meeting options for products regulated by CDER and CBER, which should help the reader select the appropriate avenue to receive the Agency’s advice and develop a viable regulatory interactions strategy.
Regulatory complexities and challenges of biosimilars
2022-06-15T10:09:00+01:00By Ash Ramzan, Manfred Kurz, Claudia Sailer
This continuing professional development (CPD) supplement focuses on the regulatory complexities and challenges associated with biosimilar products and their development. Although biosimilar products have been registered and approved for use in the EU for more than a decade, there is increasing speculation and excitement on the potential for biosimilars with increasingly complex structures, eg, multi-subunit, extensively post-translationally modified, and lipid-containing products.
The Olaratumab experience
2022-06-09T10:47:00+01:00By Alex Bloom, Natalie Thomas, Michael Sinks, Colin Gillis, Daniela Drago, Davina Stevenson
As highlighted within this CPD article, a conditional marketing authorisation (CMA) can be revoked in Europe. However only one occurrence has occurred since 2006 (in April 2019). Lartruvo (olaratumab), a humanised antibody, was granted a CMA via accelerated assessment on 9 November 2016 for adult patients with a locally advanced, ...