Expedited approval pathway had been a secondary option when traditional approval was not possible, but the CBER director wants surrogate endpoints increasingly considered from the beginning, especially with pediatric rare disease gene therapies.
Gene therapy sponsors now should expect accelerated approval to be on the table from the beginning of the development process, rather than as an alternative to a traditional pathway.
Join thousands of industry professionals who rely on Pink Sheet for daily insights
Already a subscriber?
Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.
A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.
India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.
Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.
New plan to encourage alternatives to animal testing seeks interagency cooperation to meet more aggressive targets for transformational change than the US FDA’s long-standing program.
Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.
US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth.
