- 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
– Treatment with the NaV1.8 inhibitor VX-548 led to statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the Numeric Pain Rating Scale (NPRS) – – VX-548 was generally well tolerated – – Vertex plans to advance VX-548 into pivotal
- Longer-term follow-up data demonstrate consistent and durable response to treatment - BOSTON --(BUSINESS WIRE)--Dec. 11, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from
– First-ever approval of a CRISPR-based gene-editing therapy in the U.S. – – Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment – – Multiple authorized treatment centers activated – BOSTON & ZUG, Switzerland
BOSTON --(BUSINESS WIRE)--Dec. 5, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Nancy Thornberry has been appointed to its Board of Directors as an independent director. Ms. Thornberry has more than 35 years of experience in the pharmaceutical and biotech
-Application to add ~200 non-F508del CFTR mutations to the KAFTRIO ® license- -If approved, ~2,800 people with cystic fibrosis in the European Union ages 2 and above could receive a medicine that treats the underlying cause of their disease for the first time- LONDON --(BUSINESS WIRE)--Nov.
-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
- First regulatory authorization of a CRISPR-based gene-editing therapy in the world – - CASGEVY is indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom hematopoietic
— Product revenue of $2.48 billion , a 6% increase compared to Q3 2022 — — Company raises full year 2023 product revenue guidance to approximately $9.85 billion — — U.S. FDA Advisory Committee meeting for exa-cel in SCD completed; PDUFA date for exa-cel in SCD is December 8, 2023 — — Pipeline
- New data including longer duration follow-up from SCD and TDT pivotal trials accepted for oral presentation – - Five additional abstracts accepted for poster presentation - BOSTON --(BUSINESS WIRE)--Nov. 2, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that seven