This week, at the J.P. Morgan Healthcare Conference and the Biotech Showcase, discussions underscored the growing focus on innovation in cell and gene therapies. Against this backdrop, the US Food and Drug Administration (FDA) reinforced its ongoing commitment to transformative regulatory innovation during the Cell & Gene Therapy State of the Industry (SOTI) briefing, hosted by the Alliance for Regenerative Medicine (ARM). Two key pilot programmes – Support for clinical Trials Advancing Rare disease Therapeutics (START), launched in 2023, and Collaboration on Gene Therapies (CoGenT), introduced in early 2024 – were presented as groundbreaking steps towards accelerating approval timelines for live-saving therapies targeting rare diseases and complex medical conditions. As the landscape of drug development evolves, these initiatives represent a new frontier in accelerating access to treatments that have historically faced longer regulatory pathways.
At the conference, the FDA discussed how it is engaging in a focused effort to streamline the development of rare diseases and complex medical conditions by using pilot programmes. Lessons learned from these programmes can refine future processes, potentially enabling faster and more efficient approval timelines for therapies that address significant unmet needs. The START pilot programme, launched by the FDA’s Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research, is aimed at expediting approval processes for therapies targeting rare conditions. The programme fosters enhanced communication between the FDA and sponsors, offering guidance on clinical trial designs, patient selection, and product characterisation. This initiative is particularly critical for life-threatening conditions, where delays in treatment can have severe consequences. As a pilot programme, START is not intended to replace existing regulatory pathways or establish new regulatory designations. Instead, it functions as a focused effort to learn how to streamline the development of those therapies. Lessons learned from the programme can refine future processes, potentially enabling faster and more efficient approval timelines for therapies that address significant unmet needs.
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The START pilot programme currently supports four therapies: GS-100 (Grace Science) for NGLY1 deficiency, NGN-401 (Neurogene) for Rett syndrome, rAAV-Olig001-ASPA (Myrtelle) for Canavan disease, and mRNA-3705 (Moderna) for methylmalonic acidemia. These drugs, which are currently in either Phase II or Phase III of clinical development, represent a diverse range of rare disease treatment options benefiting from enhanced regulatory guidance within the pilot. The CoGenT Global pilot programme takes a more international approach by introducing innovative global collaboration for gene therapy products targeting rare diseases – a unique approach for developers and investors in this space. The programme involves collaborating with global regulatory partners, including the World Health Organization and members of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, to explore the potential for concurrent collaborative review of gene therapy applications. By facilitating collaborative international review of gene therapy applications, the programme aims to reduce delays, facilitate cross-agency coordination, and expedite reviews for therapies that might otherwise struggle under traditional regulatory frameworks. By creating a more predictable pathway for the development and approval of gene therapy products, the CoGenT Global pilot is aiming to reduce duplication of efforts across borders, accelerating the availability of gene therapies to global patient populations.
During ARM’s SOTI briefing, Dr Peter Marks, director of the FDA, highlighted how the CoGenT pilot allows regulatory partners to participate in internal FDA meetings, share applications and supporting information, and collaborate on regulatory reviews under strict confidentiality agreements. Such measures aim to streamline global review processes and enhance the development of therapies that might otherwise struggle within traditional frameworks.
For the pharmaceutical industry, the START and CoGenT pilots represent a new and innovative opportunity to reduce timelines and costs associated with regulatory approvals. These pilots could pave the way for streamlining regulatory pathways but also enhance the global collaboration needed for tackling the complexities of rare disease treatments. With the industry looking for innovative solutions to address rare diseases, these programmes offer a promising path forward.
For more in-depth insights into other FDA regulatory initiatives and their potential impact on the rare disease and gene therapy markets, explore leading data and analytics company GlobalData’s latest report Pharmaceutical Review Designations – Trends and Industry Insights.
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