- In Europe, there are approximately 3,400 children ages 6-11 who have two copies of the F508del mutation - - Existing reimbursement agreements in countries like Ireland will enable rapid access to ORKAMBI; country-by-country reimbursement processes will now begin in other countries- LONDON
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO ® (ivacaftor) in people with cystic
-CHMP recommends Marketing Authorization for lumacaftor in combination with ivacaftor for people with cystic fibrosis ages 12 and older with two copies of the F508del mutation- -CHMP recommends Marketing Authorization for ivacaftor for children with cystic fibrosis ages 2 to 5 with 9 gating
- If approved by the European Commission , patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be
- If approved by the European Commission, nearly 300 children with cystic fibrosis and two copies of the F508del mutation would be eligible for the first time for a medicine that can treat the underlying cause of their disease - LONDON – 26/27/28 April 2023 – Vertex Pharmaceuticals (Nasdaq: VRTX)
- In Europe, there are approximately 3,400 children ages 6-11 who have two copies of the F508del mutation - BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Union Committee for Medicinal Products for Human Use (CHMP) issued a positive
- If approved, KALYDECO ® will be the first and only medicine approved in Europe to treat the underlying cause of cystic fibrosis in babies as young as 1 month with specific mutations in the CFTR gene - LONDON --(BUSINESS WIRE)--Feb. 23, 2024-- Vertex Pharmaceuticals (Nasdaq: VRTX) today announced
- If approved, people ages 12 years and older who have one copy of the F508del mutation and a gating (F/G) or residual function (F/RF) mutation will now be eligible for triple combination therapy - LONDON --(BUSINESS WIRE)--Mar. 26, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
- If approved, more than 1,500 children would be eligible for a medicine that can treat the underlying cause of their disease for the first time - LONDON --(BUSINESS WIRE)--Nov. 12, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s
-If approved, more than 1,200 children would be newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON --(BUSINESS WIRE)--Sep. 15, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee
