USA – FDA officials update on orphan drugs, gene therapies at DIA

Officials from the US Food and Drug Administration (FDA) discussed the agency’s recent efforts to support the development of products to treat rare diseases during a session at DIA’s Global Annual Meeting on Wednesday.

Orphan and rare pediatric disease designations

While the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.

Janet Maynard, director of FDA’s Office of Orphan Products Development, gave an update on FDA’s orphan drug designation program. According to Maynard, orphan drug designation requests have stabilized at just over 500 requests per year since 2016. The number of designations granted have also remained stable, in the mid-300s since 2015, with a spike to 477 in 2017.

Maynard also discussed FDA’s rare pediatric disease designation and priority review voucher program, which is set to sunset later this year without legislative action.

“Between 2013 and 2016 we saw a steady increase in terms of the number of rare pediatric disease designations requests we have received, and that number has remained greater than 50 since 2016,” she said. FDA has granted more than 50 rare pediatric disease designations each year since 2017…