The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending conditional marketing authorization of Novartis’ Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises, or pain crises, in patients with sickle cell disease aged 16 years and older.
Sickle cell disease drugs made by Novartis and Global Blood Therapeutics (GBT) may not be cost effective at current prices, a draft report published on Friday suggested, widening the debate over U.S. healthcare affordability.
South San Francisco-based Global Blood Therapeutics struck a deal with Syros Pharmaceuticals that is worth up to $375 million to discover, develop and commercialize novel therapies for the two types of blood disorders.
The U.S. Food and Drug Administration approved Oxbryta (voxelotor), a drug from Global Blood Therapeutics Inc. developed to treat sickle cell disease in adults and children 12 years or older.
Novartis AG won U.S. approval for the company’s experimental sickle cell disease drug Adakveo.
Pfizer announced that the company’s Phase III RESET clinical trial evaluating rivipansel in sickle cell disease (SCD) failed to meet the study’s primary and key secondary efficacy endpoints.
The U.S. Food and Drug Administration accepted Novartis’ Biologics License Application and granted Priority Review for the company’s investigational sickle cell medicine crizanlizumab (SEG101).
Global Blood Therapeutics reported new data from the company’s Phase III HOPE trial of voxelotor in sickle cell disease (SCD).
Novartis announced that the U.S. Food and Drug Administration granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises in patients of all genotypes with sickle cell disease.
The U.S. Food and Drug Administration lifted a clinical hold and accepted an Investigational New Drug Application (IND) for an experimental sickle cell disease treatment being jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics.