Novartis finalized a seventh new US facility as part of a $23 billion expansion. The new API site in Morrisville, North Carolina, supports end-to-end US manufacturing across multiple therapy platforms.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
The FDA issued draft guidance establishing a plausible mechanism framework for approving individualized therapies targeting ultra-rare diseases when randomized controlled trials are not feasible due to small patient populations.
The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small studies when traditional trials are not feasible.
The FDA proposed a new framework to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small studies when traditional trials are not feasible while requiring post-approval evidence collection.
