The US FDA has approved Vertex Pharmaceuticals’ (VRTX) gene therapy Casgevy (exagamglogene autotemcel) for children as young as two years old with sickle cell disease or β thalassemia.
The approval is the eighth under the Commissioner’s National Priority Voucher pilot
The US FDA has approved AbbVie’s Skyrizi (risankizumab) for use in children six years and older with moderate-to-severe plaque psoriasis or active psoriatic arthritis.
Approval was based on results from the phase 3 OptIMMize psoriasis clinical trial program.
A 55 mg pre-filled syringe has also been approved for patients weighing less than 40 kg.