Avanzanite Bioscience’s Partner Agios Announces Positive CHMP Opinion for PYRUKYND® (mitapivat) for Adults with Thalassemia

Avanzanite Bioscience B.V., a rapidly growing commercial-stage European specialty pharmaceutical company focused on rare diseases, today reported that its partner Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts focused on delivering innovative medicines for patients with rare diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the new indication for PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.

In June 2025, Avanzanite entered into an exclusive agreement with Agios to commercialize and distribute PYRUKYND across the European Economic Area, the United Kingdom, and Switzerland.

“The positive CHMP opinion for PYRUKYND represents a key step toward making this treatment available to adult patients with thalassemia – a community in critical need,” said Dr. Mark Bechter, Senior Vice President of Medical Affairs at Avanzanite. “We look forward to working with Agios to ensure access to this medicine for patients across Europe, pending approval by the European Commission,” he added.

The CHMP’s positive opinion is based on the results from the global, randomized, double-blind, placebo-controlled ENERGIZE-T and ENERGIZE Phase 3 trials in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, respectively. The European Commission will now review the CHMP’s opinion, and Agios has guided that the final decision is expected by early 2026.

About Thalassemia

Thalassemia is a rare, inherited blood disease that affects the production of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. The disease is categorized into two main types: alpha-thalassemia and beta-thalassemia, depending on which globin chain of the hemoglobin is affected. By disrupting hemoglobin production, thalassemia reduces the number of circulating red blood cells and shortens their lifespan, which leads to anemia, fatigue and serious complications.

Some individuals with thalassemia require regular transfusions (classified as transfusion-dependent thalassemia), while others only need them intermittently (classified as non-transfusion-dependent thalassemia). All patients with thalassemia experience a significant disease burden, including comorbidities, reduced quality of life and shortened life expectancy.

About ENERGIZE and ENERGIZE-T

ENERGIZE (NCT04770753) and ENERGIZE-T (NCT04770779) are global, randomized, double-blind, placebo-controlled Phase 3 trials evaluating the efficacy and safety of mitapivat in adults with alpha- or beta-thalassemia.

The ENERGIZE trial randomized 194 non-transfusion-dependent patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was the proportion of patients achieving a hemoglobin response, defined as an increase of ≥1.0 g/dL in average hemoglobin concentrations from week 12 through week 24 compared with baseline. Key secondary endpoints included changes from baseline in Functional Assessment of Chronic Illness Therapy‐Fatigue (FACIT-Fatigue) scores and in average hemoglobin concentration from week 12 to week 24. The study also assessed safety and tolerability.

The ENERGIZE-T trial randomized 258 transfusion-dependent patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was the proportion of patients achieving a transfusion reduction response (TRR), defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of transfused RBCs in any continuous 12-week period through week 48. Several additional transfusion reduction measures were included as key secondary endpoints, and achievement of transfusion independence was a secondary endpoint. The study also assessed safety and tolerability.

About Avanzanite Bioscience

Avanzanite is redefining launches of rare disease medicines across Europe. Founded in 2022 and based in Amsterdam, the Netherlands, 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 while delivering measurable impact and growth opportunities for alliance partners.

For more information, visit www.avanzanite.com.

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