- 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)
- 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
-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
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
- 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
-In Europe, approximately 12,000 people with CF ages 12 and older have two copies of the F508del mutation- -Positive CHMP Opinion was received in September 2015 - -Country-by-country reimbursement process will now begin- LONDON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
-In Europe, approximately 250 people ages 6 and older have one of 8 additional gating mutations- -KALYDECO is the first medicine to treat the underlying cause of CF in people with specific non-G551D gating mutations- EYSINS, Switzerland --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated
- European Commission approval comes two months after positive CHMP opinion - GENEVA --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has approved KALYDECO TM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who
- If approved, KALYDECO ® (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, as young as 6 months of age - LONDON --(BUSINESS WIRE)--May 1, 2020-- Vertex
- If approved, ivacaftor will be the first and only medicine to treat the underlying cause of cystic fibrosis for children this young - LONDON --(BUSINESS WIRE)--Oct. 18, 2019-- Vertex Pharmaceuticals (Europe) Limited today announces that the European Medicines Agency’s (EMA) Committee for
