FDA Announces Priority Review Vouchers for Two Pediatric Disease Therapies
The FDA issued priority review vouchers to sponsors of two rare pediatric disease therapies, United Therapeutics’ cancer drug Unituxin and Asklepion Pharmaceuticals’ Cholbam for a severe metabolic disorder.
Unituxin (dinutuximab), combined with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA), is intended to treat pediatric patients with high-risk neuroblastoma who achieved at least a partial response to prior first-line multiagent, multimodality therapy. Neuroblastoma is the most common pediatric solid tumor occurring outside the brain and the most common cancer in infants.
Cholbam (cholic acid) is used to treat bile acid synthesis disorders caused by single enzyme defects that prevent the body from producing bile acids. The drug is also an adjunctive treatment for peroxisomal disorders, including Zellweger spectrum disorders. Patients exhibit manifestations of liver disease, steatorrhea and complications from decreased fat-soluble vitamin absorption.
To qualify as a rare pediatric disease drug, the prevalence of the disease or condition treated in the U.S. must be below 200,000 patients and more than half of those must be younger than 18.
The FDA’s goal in granting a priority review designation is to act on the marketing application within six months, rather than the 10 months typical for a standard review. — Anne Wright
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