ICER Says Gene Therapies For Sickle Cell Likely To Improve Lives, But Points To Uncertainties
The drug pricing watchdog said improvement to patients’ quality and length of life from lovo-cel and exa-cel were likely, but noted ongoing uncertainty around durability and toxicity concerns.
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PTC Misses Phase III In Friedrich Ataxia, But Isn’t Giving Up
Pointing to data showing benefit in patients who completed 72 weeks of therapy, PTC thinks vatiquinone still could be viable for pediatric FA patients. Restructuring won’t affect Upstaza.
Vertex/CRISPR Are First To US FDA With A CRISPR Gene-Editing Therapy For Sickle Cell Disease
The companies completed a rolling BLA filing for exagamglogene autotemcel (exa-cel) for sickle cell disease and beta thalassemia, a decade after the discovery of CRISPR.
Investors Take Flight After Bluebird Bio’s Sickle Cell Delay
Bluebird’s first two gene therapies are on the market, but a new delay to its biggest hope, the sickle cell disease candidate lovo-cell, has spooked investors.