Key Takeaways
- US FDA's agreements with plans to seek accelerated approval of Clene’s amyotrophic lateral sclerosis drug and uniQure’s Huntington’s disease gene therapy reflect growing pressure to expand the expedited pathways for neurodegenerative diseases.
- Clene will use expanded access protocol data to support CNM-Au8’s effect on a surrogate endpoint measuring neurofilament light protein as part of a planned second quarter 2025 NDA submission.
- UniQure expects to submit a BLA for AMT-130 based on an intermediate endpoint in Phase I/II trials compared to natural history controls.
Real-world evidence from expanded access programs and a natural history external control played important roles in recent US FDA decisions on the suitability of the accelerated approval pathway for
Neurodegenerative diseases have not fit neatly into the accelerated approval pathway, as illustrated by the controversies stemming from FDA approvals of Sarepta’s ALS...
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