Vertex Pharmaceuticals
NEWS
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.
The deal follows the FDA approval of Vertex’s gene-editing sickle cell treatment and Editas’ earlier legal battle over rights to the technology.
The company’s non-opioid painkiller candidate VX-548 decreased Numerical Pain Rating Scale scores by at least two points after 12 weeks in a mid-stage diabetic peripheral neuropathy study.
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
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