Agios Pharmaceuticals, Inc. (AGIO) announced that the European Commission has approved PYRUKYND for adults with alpha- or beta-thalassemia, marking the first and only medicine authorized across all EU member states for both transfusion-dependent and non-transfusion-dependent forms of the disease.
Thalassemia is a rare inherited blood disorder that reduces the body's ability to produce healthy red blood cells, often leading to chronic anemia, fatigue, and the need for lifelong blood transfusions. Treatment options have historically been limited, especially for adults who require frequent transfusions.
PYRUKYND (mitapivat) is an oral activator of pyruvate kinase, an enzyme that helps red blood cells produce energy. By improving red blood cell function, the therapy aims to reduce anemia and lessen the burden of transfusion for patients living with this lifelong condition.
The approval is based on results from two global Phase 3 trials- ENERGIZE and ENERGIZE-T— which evaluated Mitapivat in adults with non-transfusion-dependent and transfusion-dependent thalassemia, respectively.
- ENERGIZE enrolled 194 adults and measured improvements in hemoglobin levels, with the primary endpoint defined as a =1.0 g/dL hemoglobin increase from Week 12 through Week 24.
- ENERGIZE-T enrolled 258 adults and evaluated reductions in transfusion burden, with the primary endpoint defined as a =50% reduction in transfused RBC units over any consecutive 12-week period through Week 48.
Both studies were randomized, double-blind, placebo-controlled and demonstrated clinically meaningful benefits in their respective patient groups.
"Thalassemia imposes a profound daily burden on patients, which is compounded by a lack of accessible treatment options," said Dr. Maria Domenica Cappellini, a Phase 3 investigator. She noted that the results show PYRUKYND's potential to improve outcomes regardless of genotype or transfusion status.\Agios CEO Brian Goff called the approval "an important achievement," highlighting that Mitapivat is now approved in four key markets— the U.S.,Saudi Arabia, United Arab Emirates, and EU— reinforcing the medicine's global therapeutic potential.
The company will continue its partnership with Avanzanite Bioscience B.V., which will distribute and commercialize PYRUKYND across Europe.
AGIO has traded between $22.24 and $46.00 over the past year. The stock is currently trading at $29.27 up 1.67%.
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