Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.
The proposal, purported to save $83 billion over a decade, is a "dagger in the heart of all patient advocacy nonprofits," said the head of a cancer patient support group.
The regulator approved the indication based on data showing Kisqali with an aromatase inhibitor improved invasive disease-free survival versus just an aromatase inhibitor.
NEW YORK – UniQure on Monday said it will begin treating a second cohort of Fabry disease patients with a higher dose of its AMT-191 gene therapy in a Phase I/IIa trial. The decision to advance the ...