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Hemophilia Accelerated Approval: Factor Activity Useful Surrogate, Panel Says

 
• By Grace Moser

Variability in assays is important issue in managing patients at “trough” levels, experts at US FDA hemophilia workshop suggest. However, assuming some significant level of activity is restored, the exact level isn’t overly important. 

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The US FDA’s takeaway from a recent expert panel discussion on hemophilia factor activity as a surrogate endpoint is that the measure is appropriate at a “certain threshold” even though variability exists between the assays used to measure it.

In a recent draft guidance about gene therapies for treatment of hemophilia, the agency proposed to allow utilization of factor levels as a surrogate endpoint, but noted that sponsors...

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