Recent Progress in European Advanced Therapy Medicinal Products and Beyond

Abstract

Cell- and gene-based therapies form one of the pillars of regenerative medicine. They have the potential to transform quality of life and improve the health status of patients with genetic and cellular defects, including genetic diseases, neurodegenerative diseases and tissue malignancies, amongst others. Despite numerous challenges, in the last decade, tremendous unified efforts by research and clinical scientists in academic, translational and industry settings have resulted in tangible outcomes in the form of many marketing authorizations and approved commercial firsts, such as Glybera®, Kymriah®, YESCARTA®, Holoclar®, and Luxturna™. This report presents a succinct analysis of developments in the regenerative medicine landscape, including immuno-oncology, with a focus on the European Union and examples of clinical and commercial successes and failures. The factors that led to these exciting developments in immune-oncology are also considered. Concurrently, several key issues, spanning from the identification of unmet clinical need, associated challenges, economic evaluation to policy improvements are emphasized. Furthermore, industry insights encompassing the five-dimensional research and development framework for the focused development of medicine, pricing and reimbursement issues, technology adoption and permeation of innovative advanced therapy medicinal products in the clinical set up are reflected upon, following elaborate discussions that transpired in different thematic tracks of Tissue Engineering & Regenerative Medicine International Society European Chapter 2017 Industry Symposium.

Mesenchymal Stem Cells Beyond Regenerative Medicine

“…The approval emanated from a Phase III trial reporting that Darvadstrocel led to 50% combined remission, which was maintained after 1 year of treatment, in comparison with 34% in the control arm (Panés et al, 2016(Panés et al, , 2018Panes et al, 2017). Interestingly, several "orphan designation" approvals were granted by the European commission according to certain guidelines for the use of human MSCs in the treatment of GvHD, thromboangiitis obliterans (Buerger disease), and ALS (Yu et al, 2018 According to their study, the effectiveness of MSC-FFM is due to donor selection in addition to strict collection and preparation processes (Bader et al, 2018), which yield adequate doses of MSCs with high batch-to-batch consistency (Elgaz et al, 2019). The distinguished data on MSC-FFM clearly elucidate the reasons behind the discrepancies (different survival rates and response levels to allogeneic MSC) and failures of other phase III clinical trials (Galipeau, 2013;Galipeau and Sensébé, 2018).…”

Beyond the Present Constraints That Prevent a Wide Spread of Tissue Engineering and Regenerative Medicine Approaches

“…Medicines based on genes, cells, and tissues are regulated by the same pathway and are termed as advanced therapy medicinal products (ATMPs) by EMA. They are broadly classified as gene therapy medicines (GTM), somatic cell therapy medicines (SCTM), and TEMPs (Yu et al, 2018). SCTMPs and TEMPs are distinguished on the basis of their mechanism of action.…”

Defining a Regulatory Strategy for ATMP/Aerosol Delivery Device Combinations in the Treatment of Respiratory Disease

“…Advancements in science and technology have seen the development of new therapeutic opportunities for the treatment and prevention of high-burden human diseases with unmet medical needs [ 1 ]. Advanced therapeutic medical products (ATMP) are at the forefront of innovation and are changing the therapeutic landscape for an array of human diseases [ 2 ]. ATMP is a broad term used to group three medicinal product classes intended for human use which encompass somatic cell therapy medicine, gene therapy medicines, and tissue-engineered medicines [ 3 ].…”