Regulatory Flexibility: BrainStorm’s Problematic NurOwn BLA May Be A Bridge Too Far For US FDA
Litany of deficiencies, including product quality shortcomings and a failed Phase III clinical efficacy study, may make it impossible for the agency to exercise the type of flexibility it has with the approval of other treatments for ALS and neurodegenerative diseases.
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Highly negative FDA advisory committee review and subsequent discussions with the agency made clear the only path forward was a new study, company executives said. Troubled application shows the limits of FDA’s willingness to exercise regulatory flexibility.
The US FDA has shown astonishing flexibility in approving new drugs for neurodegenerative diseases based on efficacy evidence that falls well short of historical standards. Now the agency faces the delicate challenge of demonstrating that there are still some lines it will not cross.