Vertex Pharmaceuticals
NEWS
Two surprise companies, Vertex Pharmaceuticals and Regeneron Pharmaceuticals, made GlobalData’s list with 41.4% and 21.8% market capitalization growth, respectively.
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
Positive Phase III results for VX-548 could signal a new chapter in the fight against opioid addiction, but experts say there is still room to do better.
After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.
Vertex Pharmaceuticals and CRISPR Therapeutics have secured another FDA approval for their Casgevy Cas9 gene-edited cell therapy, this time in treating transfusion-dependent beta thalassemia.
Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.
The patient deaths were deemed unrelated to Vertex’s investigational islet cell therapy, but the study will be halted while regulatory authorities and an independent data monitoring committee review the findings.
Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA’s docket this quarter.
A week after securing FDA approval, a European Medicines Agency committee has endorsed Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease and transfusion‑dependent beta thalassemia.
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