Biotechnology - Rare diseases, Kalydeco


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FDA advisory backs expanded use of Vertex’ CF drug


There was good news for US biotech firm Vertex Pharmaceuticals yesterday, as a US Food and Drug Administration…

BiotechnologyKalydecoRare diseasesRegulationUSAVertex Pharmaceuticals

Vertex leaps on positive Ph III results with lumacaftor

Vertex leaps on positive Ph III results with lumacaftor


US biotech firm Vertex Pharmaceuticals saw its share price rocket nearly 52% to $101.20 in extending…

BiotechnologyBusiness FinanceCystic fibrosisHealth Medical PharmaKalydecolumacaftorMedicinePhenolsRare diseasesRegulationResearchVertex Pharmaceuticals

FDA backs Kalydeco for eight additional mutations that cause cystic fibrosis


The US Food and Drug Administration approved biotech firm Vertex Pharmaceuticals’ supplemental New…

BiotechnologyKalydecoNorth AmericaRare diseasesRegulationUSAVertex

Vertex outlines progress on cystic fibrosis drug development


USA-based biotech firm Vertex Pharmaceuticals provided a comprehensive update on recent progress in its…

BiotechnologyKalydecolumacaftorRare diseasesResearchVertexVX-661

Vertex shoots up 60% on positive trial results for Kalydeco and VX-661


US biotech firm Vertex Pharmaceuticals' (Nasdaq: VRTX) shares rocketed 60% to $84.87 Friday morning after…

BiotechnologyKalydecoRare diseasesResearchVertexVX-661

Health Canada approves Vertex' Kalydeco; and Sanofi Pasteur's Menactra


Vertex Pharmaceuticals (Nasdaq: VRTX) says that Health Canada has approved Kalydeco (ivacaftor), the…

BiotechnologyKalydecoMenactraNorth AmericaPharmaceuticalRare diseasesRegulationSanofiSanofi PasteurVaccinesVertex

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