AMSTERDAM — Avanzanite Bioscience has entered an exclusive agreement with Agios Pharmaceuticals to commercialize and distribute PYRUKYND® (mitapivat) across the European Economic Area, the UK, and Switzerland, the companies announced Tuesday. The partnership expands access to the first and only approved oral therapy for adults with pyruvate kinase (PK) deficiency, a rare genetic blood disorder.
Under the deal, Avanzanite will also hold rights to potential future indications as Agios advances its clinical programs in other rare hematologic diseases, including thalassemia and sickle cell disease.
“This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe,” said Adam Plich, Founder and CEO of Avanzanite. “We provide biotech partners with a capital-efficient path to sustainable patient access—without the burden of building a costly local infrastructure.”
PYRUKYND® was approved by the European Commission and the UK’s Medicines and Healthcare products Regulatory Agency as a treatment for PK deficiency, a condition that leads to chronic anemia, fatigue, and serious complications due to accelerated red blood cell breakdown. Until recently, there were no approved therapies for the condition.
Avanzanite, which currently markets two rare disease medicines, has seen rapid growth, tripling its revenue year-over-year in Q1 2025. The company plans to expand into 32 countries across Europe over the next 12 months, including new markets such as France, Italy, Romania, Spain, and the UK.
“Avanzanite goes truly pan-European,” said Plich. “And as we expand across the continent, our promise stays the same—making sure no patient is left behind.”
