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

06 Dec 2023

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Barrier — An FDA draft guidance has become a barrier to developing drugs for MPS III, advocates and sponsors say.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

Patient advocates, sponsors and clinical experts are protesting what they view as generally unworkable recommendations from the US Food and Drug Administration on development approaches and clinical trial design for new therapeutics for Sanfilippo syndrome.

Key Takeaways

FDA’s 2020 draft guidance on development of treatments for MPS III recommends randomized controlled trials, which disease community representatives say are infeasible.

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is an ultra-rare enzyme defect disease that affects young children....