With Sickle Cell Results For Pyrukynd, Agios Enters Increasingly Competitive Market
The company has significantly built up its pipeline since divesting its oncology business to Servier, with multiple Phase III readouts for Pyrukynd expected in 2024-2025.
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The company hopes to position mitapivat as an oral treatment for patients across the thalassemia spectrum. A Phase III trial in non-transfusion-dependent alpha- or beta-thalassemia was positive.
The first gene editing medicine and gene therapy for sickle cell disease were approved as the ASH meeting got under way, but novel oral drugs may be key to making treatment more accessible.
Servier’s targeted therapy, vorasidenib, can effectively delay the need for more drastic treatment in patients with low-grade glioma, but increases in liver enzymes including two cases of Hy’s law have cast some shade over the INDIGO data.