transfusion-dependent beta thalassaemia
Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE
The rare inherited disease affects approximately 11,000 people in the UK
Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval
The rare inherited disease affects more than 109,000 people worldwide
GSK’s Blenrep combination recommended by NICE to treat multiple myeloma
The decision means that eligible patients in England will be the first in the world to access the combination
NICE recommends Gideon Richter’s Ryeqo as first daily pill for endometriosis
Around 1,000 patients could benefit from the relugolix combination therapy every year
Vertex’s triple combination cystic fibrosis therapy Alyftrek approved by MHRA
Over 11,000 people in the UK are affected by the rare genetic disease
Amgen’s Blincyto recommended by NICE for new adult leukaemia indication
Up to 50% of acute lymphoblastic leukaemia patients will relapse after initial treatment
NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease
About 15,000 people in England are living with the inherited blood disorder
AstraZeneca receives NICE recommendations for lung cancer drugs Tagrisso and Imfinzi
More than 49,000 people are diagnosed with lung cancer in the UK each year
NICE recommends Santhera’s Duchenne muscular dystrophy drug Agamree
Approximately 1,700 patients with the muscle-wasting disorder in England are expected to benefit from the decision
NICE recommends Immunocore’s Kimmtrak as first drug for aggressive eye cancer
More than 100 patients are expected to be eligible for the treatment every year
NICE recommends Blueprint Medicines’ Ayvakyt to treat rare blood disorder
Systemic mastocytosis affects approximately one in every 10,000 people in the UK
Pfizer’s Elrexfio recommended by NICE to treat relapsed and refractory multiple myeloma
Approximately 5,000 people are diagnosed with the disease every year in England