Post Thumb

FDA Approves New Orphan Drug to Treat Rare Autosomal Recessive Disorder

Share it

“Today’s approval and rare pediatric disease priority review voucher underscore the FDA’s commitment to making treatments available to patients with rare diseases,” said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research.

“Prior to Xuriden’s approval, patients with this rare disorder had no approved treatment options.” The FDA granted Xuriden orphan drug designation because it treats a rare disease.

Orphan drug designation provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development.

An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment.

The manufacturer of Xuriden was granted a rare pediatric disease priority review voucher – a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

No side effects were observed in patients treated with Xuriden for up to nine months.


Article originally posted at

Post Author: Meghan Rosen

Leave a Reply

Your email address will not be published. Required fields are marked *