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Date Title and Summary Additional Formats
Toggle Summary European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia may be eligible for treatment - LONDON --(BUSINESS WIRE)--Feb. 13, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has
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Toggle Summary European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years
- More than 1,500 children now eligible to receive a treatment targeting the underlying cause of cystic fibrosis for the first time - LONDON --(BUSINESS WIRE)--Jan. 11, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for
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Toggle Summary European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis in People Ages 12 Years and Older
– For the first time, up to 10,000 people in Europe ages 12 years and older with one F508del mutation and one minimal function mutation will be eligible for a medicine that treats the underlying cause of cystic fibrosis – – People 12 years of age and older who have two F508del mutations will also
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Toggle Summary European Commission Approves KAFTRIO® in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5
-More than 1,200 children are newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON --(BUSINESS WIRE)--Nov. 23, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion
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Toggle Summary European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old
-Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease- LONDON --(BUSINESS WIRE)--Jul. 5, 2023-- Vertex Pharmaceuticals today announced that the European Commission has
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Toggle Summary FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- First CRISPR gene-editing filings to be accepted for review by FDA - - FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) – - PDUFA target action date of December 8, 2023 , for SCD and March 30, 2024 , for TDT –
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Toggle Summary FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment
-FDA Grants Priority Review of the application and sets a PDUFA target action date of March 19, 2020 - -Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages 12 and older with one F508del mutation and one minimal function mutation and in
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Toggle Summary FDA Accepts Vertex’s Supplemental New Drug Applications for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Additional CFTR Mutations
- More than 600 people with certain rare CF mutations could become newly eligible for TRIKAFTA, SYMDEKO or KALYDECO - BOSTON --(BUSINESS WIRE)--Sep. 1, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental
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Toggle Summary FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration's Pulmonary Allergy Drugs Advisory Committee (PADAC) voted 13-2 to recommend approval of KALYDECO ® (ivacaftor) in people with cystic fibrosis (CF) ages 6 and
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Toggle Summary FDA Advisory Committee Unanimously Recommends Approval of Telaprevir for People with Hepatitis C
CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Antiviral Drugs Advisory Committee to the U.S. Food and Drug Administration (FDA) voted unanimously to recommend FDA approval of telaprevir for people with genotype 1 chronic hepatitis
HCV
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