PYRUKYND® (mitapivat) is a first-in-class pyruvate kinase (PK) activator, approved by the European Commission for adults with PK deficiency
The agreement includes commercialization and distribution rights for potential future indications
Furthered by this partnership with Agios, Avanzanite is setting a new paradigm in biotech commercial and distribution partnerships, and in how orphan medicines reach patients across Europe

Avanzanite Bioscience B.V. ("Avanzanite"), a fast-growing commercial-stage specialty pharmaceutical company dedicated to bringing rare disease medicines to patients across Europe, announced today an exclusive agreement with Agios Pharmaceuticals Inc. (NASDAQ:AGIO), a Boston-based biotech company focused on the development and commercialization of rare disease medicines. Under the agreement, Avanzanite will commercialize and distribute PYRUKYND® (mitapivat) across the European Economic Area, the UK and Switzerland.

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Adam Plich, Founder & CEO

"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. We look forward to working with Agios to help bring PYRUKYND® to patients across Europe, further advancing our efforts to set a new benchmark for biotech commercial and distribution partnerships in the region. This is just the beginning."

PYRUKYND®, a first-in-class, oral, pyruvate kinase (PK) activator, is approved by the European Commission and the UK's Medicines and Healthcare products Regulatory Agency for the treatment of adult patients with PK deficiency. PK deficiency is an ultra-rare, inherited condition that causes premature red blood cell breakdown, leading to chronic anemia, serious complications, and reduced quality of life. Until recently, there were no approved treatments. Agios also has a robust mid- and late-stage pipeline, with clinical programs focused on other rare diseases, including thalassemia and sickle cell disease. The partnership also includes potential future indications.

Avanzanite is rapidly scaling its operations, having tripled its revenue in Q1 2025 year-over-year, with two rare disease medicines already on the market. Over the next 12 months, the company will expand into 32 European countries, 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."

About Avanzanite

Avanzanite is redefining launches of rare disease medicines across Europe. Founded in 2022 and based in Amsterdam, the company partners with biotech innovators to unlock the full commercial value of orphan medicines continent-wide. With our deep expertise in market access, we navigate Europe's complex landscape like master chess players – ensuring no patient is left behind.

Learn more at www.avanzanite.com.

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Avanzanite Bioscience B.V.

Phone: +31 20 301 21 13

Email: media@avanzanite.com

For medical enquiries, contact medinfo@avanzanite.com