The idea of a biotech “platform” company is almost as old as the biotech sector itself.
US FDA’s ‘Platform’ Pathway Fits For LNPs And siRNA But Not Delivery Devices
The US FDA’s new guidance defining a “Platform Technology Designation” makes clear that what counts as a “platform” may not match industry’s use of the term.
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The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
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Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.
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The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.
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Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
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Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.
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The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.
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A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.