- 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 –
-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
-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
– 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
- 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
- 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
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated today announced that David Altshuler , M.D., Ph.D., will join the company as Executive Vice President, Global Research and Chief Scientific Officer. Dr. Altshuler was one of the four founding members of the Broad Institute of Harvard
- 4.54 log 10 median reduction in HCV RNA after 7 days of dosing; ALS-2200 was well-tolerated with no serious adverse events and no discontinuations due to adverse events - - Vertex moving forward with multiple Phase 2 trials in early 2013 exploring all-oral combinations, including VX-135 with
- New data at North American CF Conference showed a reduction in sweat chloride when KALYDECO was added to VX-809 in people with the F508del mutation — - Part 2 of study now enrolling patients - ANAHEIM, Calif. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced
- 90% of people with the ‘CC' variation of IL28B who were new to treatment and received a telaprevir-based regimen achieved a viral cure, 78% of them were eligible to stop all treatment at 24 weeks - - Nearly three-fold improvement in viral cure rates was observed among people with the ‘CT' and
