EU approves Oxbryta for hemolytic anemia due to sickle cell disease – Global Blood Therapeutics

Global Blood Therapeutics announced the European Commission (EC) has granted Marketing Authorization for Oxbryta (voxelotor) for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Oxbryta, a once-daily, oral treatment, is the first medicine approved in Europe that directly […]

Experimental gene therapy reverses sickle cell disease for years

A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients. Four patients at NewYork-Presbyterian/Columbia University Irving Medical Center participated in the multicenter study, the first to report on such […]

NICE (UK) recommends Adakveo as a treatment for preventing recurrent sickle cell crises – Novartis

Novartis is pleased to announce that eligible patients in England and Wales will soon have routine access to Adakveo (crizanlizumab) under a Managed Access Agreement (MAA). The news comes as the National Institute for Health and Care Excellence (NICE), published the Final Appraisal Determination (FAD) recommending crizanlizumab as an option for preventing recurrent sickle cell crises (two or more vaso-occlusive […]