Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance

 
• By Sue Sutter

Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

Barrier
An FDA draft guidance has become a barrier to developing drugs for MPS III, advocates and sponsors say. • Source: Shutterstock

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