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 Grants Priority Review for Therapy for Life-threatening Hereditary Immune Disease
February 23rd 2023If approved, pozelimab would be the first treatment for CHAPLE disease, which causes damage to blood and lymph vessels along the upper digestive tract. The target action date for the FDA decision is Aug. 20, 2023.