The Cure Sanfilippo Foundation is encouraging the U.S. Food & Drug Administration (FDA) to consider Sanfilippo syndrome as a candidate for the agency’s accelerated approval pathway. According to a recent foundation-led study, families of Sanfilippo children are willing to accept modest treatment benefits even if they come with risks.
“Doing nothing is the biggest risk in Sanfilippo!” Glenn O’Neill, foundation president and father to a Sanfilippo child, said in a press release. His statement came at the end of a presentation Oct. 20 at the 13th Annual Rare Disease Scientific Workshop.
The FDA’s Accelerated Approval Program allows for earlier approval of therapies that treat serious conditions and fill an unmet medical need. Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a rare genetic disorder characterized by the mutations in genes that encode enzymes that break down the complex sugar molecule known as heparan sulfate.
