Avanzanite Bioscience B.V., a commercial-stage specialty pharmaceutical company, has announced an exclusive partnership with Agios Pharmaceuticals Inc. (NASDAQ:AGIO) to commercialize and distribute PYRUKYND® (mitapivat) across the European Economic Area, the UK, and Switzerland. The agreement represents a significant milestone in bringing the first-in-class treatment for pyruvate kinase deficiency to European patients.
First-in-Class Treatment for Ultra-Rare Disease
PYRUKYND® is an oral pyruvate kinase (PK) activator that has received approval from both the European Commission and the UK's Medicines and Healthcare products Regulatory Agency for treating adult patients with PK deficiency. The condition is an ultra-rare, inherited disorder that causes premature red blood cell breakdown, resulting in chronic anemia, serious complications, and reduced quality of life.
"Until recently, there were no approved treatments" for PK deficiency, highlighting the critical unmet medical need that PYRUKYND® addresses. The drug's approval marks a breakthrough for patients who previously had limited therapeutic options for managing this debilitating condition.
Strategic Partnership Model
Adam Plich, Founder and CEO of Avanzanite, emphasized the partnership's significance in transforming rare disease therapy access in Europe. "This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe," Plich stated. "We provide biotech partners with a capital-efficient path to sustainable patient access – without the burden of building a costly local infrastructure."
The collaboration extends beyond PYRUKYND®'s current indication, with the agreement including commercialization and distribution rights for potential future indications. Agios maintains a robust mid- and late-stage pipeline with clinical programs focused on other rare diseases, including thalassemia and sickle cell disease.
Rapid European Expansion
Avanzanite is experiencing significant growth, having tripled its revenue in Q1 2025 year-over-year with two rare disease medicines already on the market. The company has outlined ambitious expansion plans, targeting 32 European countries over the next 12 months, including new territories of Italy, France, the UK, Romania, and Spain.
"Avanzanite goes truly pan-European," concluded Plich. "And as we expand across the continent, our promise stays the same – making sure no patient is left behind."
Company Background
Founded in 2022 and based in Amsterdam, Avanzanite positions itself as redefining launches of rare disease medicines across Europe. The company partners with biotech innovators to unlock the full commercial value of orphan medicines continent-wide, leveraging deep expertise in market access to navigate Europe's complex regulatory landscape.
The partnership with Agios, a Boston-based biotech company focused on rare disease medicine development and commercialization, represents what Avanzanite describes as "setting a new paradigm in biotech commercial and distribution partnerships, and in how orphan medicines reach patients across Europe."
