The globe is anticipating an avalanche of new and novel regenerative medicine (RM) treatments, reveals Australia’s Regenerative Medicine Global Pipeline Tracker, and Australia may share in access to some.
The inaugural report, released by the Regenerative Medicine Catalyst Project Consortium, horizon-scanned the global pipeline of products in this emerging field of medicine, and postulates which might come to Australia for patient access.
While there are currently six RM therapies already approved in Australia [1], the new data shows a rich pipeline with 140 therapies in late-stage development globally in multiple therapeutic areas, and up to nine may reach Australian patients in the next five years.
The growth seen in RM is driven by an increasing prevalence of chronic diseases, genetic disorders, and cancer, coupled with increasing investments and collaboration with the medical research sector to develop therapies that will treat a variety of diseases across multiple therapeutic areas.
Analysis of the global pipeline includes therapies that are already registered overseas as well as those that are likely to get to market through global clinical development pipelines, and subsequently to the Australian market.
The cell and gene therapies (CGT) pipeline comprises cell therapies, gene therapies, and gene-modified cell therapies, and the Global Pipeline Tracker discovered that around 70 percent of products in this pipeline are in phase III of development, 11 percent of products are already undergoing regulatory review (mostly in major developed markets, such as the US and EU), and that Phase II/III accounted for 16 percent of the pipeline.
The analysis of CGT products, looks at the pipeline and determines those most likely to enter the Australian market in the next five years and the timelines for entry. Likelihood to enter the Australian market was based on estimates of the likelihood of approval, companies’ current presence in Australia and clinical trials of the product in Australia. Timelines for entry were based on RM approved products average timings of phase III completion, phase III to regulatory filing, filing to approval and average approval gap US vs Australia.
Based on these estimates the following products were considered to be the most likely to enter the Australian market:
- Cell Therapy: StrataGraft® (Mallinckrodt Plc) and RVT-802 (Enzyvant Sciences Ltd) in 2023; Omidubicel (Gamida Cell Ltd) and Stapuldencel-T (Sotio AS) in 2024;
- Gene Therapy: Vutrisiran (Alnylam Pharmaceuticals Inc) in 2023; Fitusiran (Sanofi) and Etranacogene dezaparvovec (UniQure NV) in 2025; and
- Gene Modified Cell Therapy: Lisocabtagene maraleucel (Juno Therapeutics Inc) in 2023; Ciltacabtagene autoleucel (Legend Biotech Corp) in 2024.
Tissue engineered products make up 43 of the 140 products in development globally, and are typically approved through a medical device regulatory pathway. Musculoskeletal disorders are the leading therapy area for these products, followed by dermatology and cardiovascular diseases.
The future of RM is bright: given the quantity of early-stage clinical trials already underway it is expected that the number of late-stage pipeline products will also continue to rise, ultimately meaning that even more RM treatments are expected to be approved in the future. The opportunity of regenerative medicine technology is being recognised and with a greater number of smaller biotech companies entering into this space, even more options will become available. As the RM market becomes more established, both small biotech companies and multinational pharma firms are seeking to get a larger share of the market, and so are entering into strategic alliances or acquiring small- to mid-size players or products.
Download the Australia’s Regenerative Medicine Global Pipeline Tracker report here.
About the RM Catalyst Project
The RM Catalyst’s seven partners hold extensive insight and experience in the life science and regenerative medicines landscape in Australia. Led by AusBiotech, partners include Medicines Australia, Cell Therapies Pty Ltd, Novartis Australia and New Zealand, Biointelect Pty Ltd, Research Strategies Australia, and MTPConnect.
The RM Catalyst has received matched funding through MTPConnect’s Growth Centre Project Fund Program, an Australian Government initiative supported by the Department of Industry, Science, Energy and Resources. It is a competitive matched funding program that aims to invest in ideas to boost the innovation, productivity and competitiveness of Australia’s MTP sector.
The project is developing nine reports to create the foundations for an RM future for Australia, these include:
- The comprehensive Regenerative Medicine Value Chain report considers the chain of activities involved in RM therapy development;
- Australia’s Regenerative Medicine Clinical Trials Database benchmarks Australia’s clinical trials and captures the portion in RM, and seeks to categorise them into type and phase;
- Australia’s Regenerative Medicine Manufacturing Capacity & Capability provides a new evidence base and model against which to map, build and strengthen our sovereign GMP manufacturing position over time; and
- Australia’s Regenerative Medicine Catalyst Body Funding & Model Structure provides a recommendation for a funding and model structure for the Regenerative Medicine (RM) sector ‘catalyst’ collaboration body, with a scope that includes industry as a key component within a broader sector.
Read the other reports and more about the Regenerative Medicine Catalyst Project here.
