September 29th 2023
The two-component therapy of Pombiliti plus Opfolda to treat adults living with late-onset Pompe disease will have an annual list price of $650,000.
FDA Accepts sBLA for Evkeeza for Children with Rare High Cholesterol
November 30th 2022If approved, Evkeeza would be the first treatment to help children as young as 5 years old control high LDL cholesterol caused by homozygous familial hypercholesterolemia. The FDA target action date is March 30, 2023.