Challenges and Emerging Strategies in ATMPs

Advanced therapy medicinal products (ATMPs) are a significant innovation in medicine, categorized by the European Medicines Agency into four types: gene therapy medicines, somatic cell therapy medicines, tissue-engineered medicines and combined ATMPs. They hold potential for treating various conditions, including rare diseases, degenerative diseases like Parkinson’s and Alzheimer’s and cancers. The global market for ATMPs was valued at US$9.28 billion in 2023 and is projected to reach US$22.80 billion by 2032, growing at a compound annual growth rate of 10.50%. Gene and cell therapies are advancing rapidly, with over 1,700 approved clinical trials worldwide. Notable successes include Glybera for lipoprotein lipase deficiency and Roctavian for haemophilia. Despite limited current approvals, many gene therapies are expected to gain market approval soon. Challenges include stringent regulatory requirements, safety and ethical concerns. For example, Glybera faced multiple rejections before approval due to safety issues. Current clinical studies are exploring immune-based therapies like CAR-T, cytotoxic T lymphocytes, natural killer cells and mesenchymal stromal cells. CAR-T cell therapies, such as Yescarta and Kymriah, have shown promise in treating B-cell lymphoma. However, high development and manufacturing costs limit accessibility, exemplified by Hemgenix, a gene therapy for haemophilia B priced at US$3.5 million. Increased ATMP sales have spurred investor interest in R&D, leading to advancements in technology and manufacturing processes. Despite challenges, the future is promising with more academic and commercial ATMP clinical trials. There are now 32 approved gene therapies globally. To attract industries, the focus should shift from rare to common diseases, and alternative reimbursement models could help manage high costs. A trained workforce and public involvement are crucial for the successful delivery of ATMPs.