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Regulatory Challenges for Gene Delivery
Published in Yashwant Pathak, Gene Delivery, 2022
Vineet Mahajan, Shruti Saptarshi, Yashwant Pathak
In the EU gene therapies are classified as, advanced therapy medicinal product (ATMPs), under the EU legislation, and are governed by the ATMP Regulation (Directive 2001/83/EC, as amended by Regulation [EC] 1394/2007)35 The European Medical Association’s (EMA) Committee for Advanced Therapies (CAT) oversees the regulatory affairs of ATMPs after consultation with the European Commission. The principal EU guideline (under revision) is the basis for development of gene therapies. It provides a thorough strategic plan for gene therapy product development, covering quality, non-clinical, and clinical aspects of the ATMPs. Unlike the regulations for other biotech-based medicinal products, the technical requirements of quality or clinical data required to demonstrate safety of the ATMP product may be highly specific. These guidelines are targeted at mitigating risks to the beneficiaries of the ATMP treatments, once the products are authorized for use. Following points provide details of the EU regulatory guidelines for ATMPs:
Advanced Cell Therapy for Asherman's Syndrome
Published in Carlos Simón, Carmen Rubio, Handbook of Genetic Diagnostic Technologies in Reproductive Medicine, 2022
Jordi Ventura, Xavier Santamaria
In summary, this study demonstrated that the use of CD133+ BMDSCs can be useful in the treatment of AS and other endometrial diseases. Indeed, the European Medicines Agency (EMA) as well as the US Food and Drug Administration (FDA) have acknowledged these results and have issued a positive opinion to consider CD133+ cells as the first Orphan Drug Designed (ODD) therapy for the treatment of AS, categorizing these cells as Advanced Therapy Medicinal Product (ATMP); supervised phase I/II and III trials are currently being performed. Obviously, further developments need to be performed to determine optimal dosage, to achieve a long-term follow-up, and to carry out a randomized trial, but this can be considered to be the first focused proof-of-principle study. Currently, a phase I/II trial is being conducted in order to assess long-term safety and optimal dosage as well as to determine different MoAs.
Big promise, big business
Published in Christine Hauskeller, Arne Manzeschke, Anja Pichl, The Matrix of Stem Cell Research, 2019
Claire Tanner, Alan Petersen, Casimir MacGregor, Megan Munsie
Despite initially being banned in the Netherlands (Sheldon, 2007), X-Cell was able to successfully operate due to ambiguity in German and EU regulations. In particular, there was ambiguity with respect to whether the services offered by X-Cell should be considered a medical practice or regulated as a therapeutic product. Notably, under European law doctors are allowed to treat patients in European hospitals, under a ‘hospital exemption’, when a therapy is in the process of being certified, clinically tested or used as a treatment of last resort, subject to the approval of a relevant federal authority. Furthermore, whilst it could be considered that the SCTs administered at X-Cell were an Advanced Therapy Medicinal Product (ATMP) and therefore under the remit of the European Medicine’s Agency and relevant national authorities, this legislative change did not come into effect in Germany until December 2008. The thirty-six-month transition period contributed to confusion as to whether a stem cell-based product had to be regulated under the EU regulation or German Drug Law (Schnitger, 2014). There was also significant debate around the definition of a stem cell-based product which compounded existing jurisdictional and regulatory uncertainty around the practices of X-Cell (Petersen et al., 2017, 103–105).
10th antibody industrial symposium: new developments in antibody and adoptive cell therapies
Published in mAbs, 2023
Ana Antunes, Luis Alvarez-Vallina, Federico Bertoglio, Nicolas Bouquin, Stéphanie Cornen, Francis Duffieux, Pierre Ferré, Raphaëlle Gillet, Christian Jorgensen, Mark B Leick, Bernard Maillère, Hélène Negre, Mireia Pelegrin, Nicolas Poirier, Dietmar Reusch, Bruno Robert, Guy Serre, Alain Vicari, Martin Villalba, Christoph Volpers, Gavin Vuddamalay, Hervé Watier, Thierry Wurch, Lennart Zabeau, Stefan Zielonka, Baolin Zhang, Alain Beck, Pierre Martineau
Dr. Manel Juan (Head of the Immunology Service, Hospital Clínic de Barcelona, Spain) described the generation of a new autologous CAR targeting CD19 (ARI-0001). This CAR obtained similar clinical results than commercial CARs used in the same clinical settings and was approved by the Spanish Agency of Medicines and Medical Devices (AEMPS). ARI-0001 was granted a hospital exemption to be used as advanced therapy medicinal product (ATMP), leading to its marketing authorization under specific conditions. These are: the exemption is only applicable to individual patients treated in the hospital (mainly academic centers that developed the ATMP and are supported by the national competent authority and limited to European Union (EU) member states; the exemption is intended for >25-year-old patients with relapsed or refractory CD19+ acute lymphoblastic leukemia. This authorization is the first step in the development of and access to completely academic CAR T-cell products in the EU and can be used as an intermediate step before obtaining a centralized marketing authorization by the European Medicines Agency (EMA). The cooperation between academic partners, who must follow strict standards of traceability, pharmacovigilance, and quality, should favor the accessibility of this product and the approval of other academic ATMPs. M. Juan’s team is completing a clinical trial on an anti-B-cell maturation antigen (BCMA) CAR-T for multiple myeloma, with a solid clinical response.12
Pricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact
Published in Expert Opinion on Orphan Drugs, 2022
As we could not have accessed up-to-date data after 2017, the analysis of this study was based on data related to ODs approved in South Korea over a long period (2007–2017). Therefore, we could not include the big issue of reimbursement for high-expensive Advanced Therapy Medicinal Product (ATMP) OD approved after 2017. While a comparison of the analysis of the financial impact of South Korea’s new ODs to other countries provided very useful information, the wider scope of the research has not been addressed in this study. Additionally, the budget impact of ODs can only be accurately calculated when the orphan indication is derived during the budget impact for ODs with a multi-indication, and when the refund rate is taken into account in the analysis when a refund type RSA has been signed. However, claim data based on indications could not be established for South Korea and refund rates could not be taken into account as they are confidential data.
Making COVID-19 mRNA vaccines accessible: challenges resolved
Published in Expert Review of Vaccines, 2022
The mRNA products are listed by EMA under its advanced therapy medicinal product (ATMP) program [36,37]. ATMPs can be classified into three main types: gene therapy medicines, somatic cell therapy, and tissue-engineered medicines. An equivalent program at the FDA is the regenerative medicine advanced therapy (RMAT) classification [38] that includes cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except human cells, tissues, cellular and tissue-based products. Additionally, this designation also includes if the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.