Vertex Pharmaceuticals
NEWS
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.
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.
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