Earlier this year, ISCT published their inaugural regulatory report, offering insights into the global cell and gene therapy (CGT) regulatory landscape and the latest approvals. Produced in collaboration with Citeline, the report – which focuses on cell therapies, genetically-modified cell therapies, and gene therapies – highlights changes in legislative frameworks, and subsequent impact on the CGT industry.
Here, we have summarized our key takeaways from this year’s report . . .
Genetically-modified cell therapies are on the rise
Year-on-year, cell therapies continue to maintain their dominance, constituting a significant 71% of all products approved globally.
Within this, there is a clear trend for genetically-modified cell therapies, with an impressive pipeline of 1150 therapies currently in development, surpassing the number of both non-genetically modified cell therapies (839), and gene therapies (920).
Of these, 804 genetically-modified cell therapies are currently in the pre-clinical phase of development. With remarkable momentum, these modified therapies are seemingly leading the way in shaping the future of CGT.
Update on approvals
The latest CGT approvals are significant milestones in the advanced therapies landscape. As of June 1st 2023, 87 advanced therapy products have been approved globally, with 62 attributed to non-genetically modified cell therapies, 11 to genetically-modified cell therapies, and 14 to gene therapy products.
Among them are:
- Hemgenix® – an adeno-associated viral vector based gene therapy, which offers treatment for adults with Hemophilia B
- Ebvallo® – the first cell therapy to treat transplant patients with post-transplant lymphoproliferative disease
- Vyjuvek® – the first topical gene therapy for the treatment of wounds in patients with Dystrophic Epidermolysis Bullosa
- Omisirge® – a cell therapy for patients with blood cancers to reduce the risk of infection following stem cell transplantation
- Carvykti® – a cell therapy for patients with relapsed or refractory multiple myeloma, earns approvals from FDA, EMA, MHRA, PMDA, and TGA
Key cell therapies under regulatory review
14 genetically-modified cell therapies are currently under regulatory review globally. Of these, nine are in Phase III clinical trials, and five have completed clinical trials and are now awaiting registration or approval by a governing body before they can be made commercially available to patients.
These five therapies are:
- CARsgen Therapeutics – focusing on multiple myeloma for patients in China
- Nanjing IASO Biotherapeutics – also dedicated to multiple myeloma for patients in China
- CRISPR Therapeutics – focusing on anaemia, sickle cell, and thalassaemia for patients in the US, EU and the UK
- Juventas Cell Therapy – targeting leukaemia and acute lymphocytic conditions for patients in China
- Bluebird Bio – focusing on anaemia and sickle cell treatments for patients in the US
Global legislative and framework changes
Recent efforts by global organizations like the FDA and MHRA, have played a pivotal role in shaping the legislative and policy frameworks to improve the accessibility and affordability of CGT products.
- Food and Drug Administration (FDA): The FDA has published a discussion paper titled “Distributed Manufacturing (DM) and Point-of-Care (PoC) Manufacturing of Drugs”, identifying challenges presented by DM and POC, and posing key questions to facilitate public comment. The paper intends to gather feedback from the public and address key questions to shape future policy development.
- World Health Organization (WHO): Simultaneously, the WHO has released an early draft of their whitepaper outlining their approach to developing a global regulatory framework for cell and gene therapy products, reflecting their commitment to fostering revolutionary medical advancements on a global scale.
- European Health Union: In a significant step forward, the EU is proposing to reform pharmaceutical legislation, with a focus on expanding the availability, accessibility, and affordability of medicines. By implementing a simplified regulatory framework, this proposal aims to promote innovation and foster competitiveness in the pharmaceutical industry, driving advancements in medical treatments.
- Medicines and Healthcare products Regulatory Agency (MHRA): The MHRA in the UK is taking a progressive approach by introducing a first-of-its-kind framework that streamlines point of care manufacturing of innovative medicines. This initiative is set to benefit patients who currently lack viable treatment options, as it opens new avenues for accessing innovative and cutting-edge medicines.
The CGT industry is undergoing a pivotal transition. As promising therapies navigate the regulatory landscape, global regulatory authorities are working to create a more progressive environment – fostering not only the rapid advancement of groundbreaking treatments, but also their accessibility. The trends highlighted in ISCT’s report underscore CGTs’ immense potential to bring about a paradigm shift in healthcare, promising a future where innovative therapies are not just approvable but accessible and affordable too.
