Sanofi to Spend $245 Million for Rare Pediatric Disease Voucher
French drug giant Sanofi will spend $245 million to acquire San Diego-based Retrophin’s rare pediatric disease priority review voucher.
Sanofi has no less than 13 medicines in its late-stage pipeline, spokeswoman Mary Kathryn Steel says, and this voucher will help deliver one of those to the market faster. The companies announced the agreement last week.
Retrophin received the voucher in March when it purchased the rights to Asklepion Pharmaceuticals’ Cholbam (cholic acid), which the FDA had just approved for children and adults with single-enzyme bile acid disorders and peroxisomal disorders. It marked the third and final voucher to be awarded by the FDA, triggering the program’s March 2016 sunset unless Congress reauthorizes it.
The U.S. House’s 21st Century Cures Act would allow the FDA to again hand out rare pediatric disease vouchers, but only until December 2018. Lawmakers on the Energy and Commerce Committee have said they hope to get the bill to President Barack Obama’s desk by the end of the year.
Retrophin’s pipeline contains two other drugs for rare diseases: A therapy for focal segmental glomerulosclerosis is in Phase II, while another for pantothenate kinase-associated neurodegeneration has not yet reached human testing.
The rare pediatric disease priority review voucher grants a subsequent priority review for companies sponsoring applications for rare childhood diseases. The FDA awarded the first voucher to BioMarin in February 2014 for its Morquio A Syndrome therapy Vimizim (elosulfase alfa). United Therapeutics won the second voucher in March, with approval of Unituxin (dinutuximab) for neuroblastoma. — Lena Freund