Annora's Generic Glycerol Phenylbutyrate Receives Approval in the U.S.
Two new gene therapies—from Novartis and Spark Therapeutics—carry price tags higher than any other drugs in the U.S., elbowing aside the brands we've seen at the top for years, a GoodRx analysis shows. And with drugmakers doubling down in rare diseases, the trend will likely continue.
Horizon Pharma plc Announces FDA Approval to Expand the Age Range for RAVICTI® (glycerol phenylbutyrate) Oral Liquid to Include Newborns
Rare disease group Swedish Orphan Biovitrum (Sobi) says it has gained NHS England reimbursement for Ravicti for long-term management of urea-cycle disorders (UCDs) in adults and children.
RAVICTI® (glycerol phenylbutyrate) : Horizon Therapeutics v. Par Pharmaceutical
After a three-month review delay at the FDA, BioMarin Pharmaceutical on Thursday won the agency’s backing for its orphan drug Brineura, now approved to treat the ultrarare CLN2 disease.