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AMSTERDAM - The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for PYRUKYND (mitapivat) to treat anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, according to a press release from Avanzanite Bioscience. This development comes as InvestingPro data shows Agios Pharmaceuticals (NASDAQ:AGIO) maintaining a strong financial position with more cash than debt on its balance sheet and impressive revenue growth of 30.57% over the last twelve months.
The oral pyruvate kinase activator is already approved in Europe for adults with pyruvate kinase deficiency. The European Commission will now review the CHMP’s recommendation, with a final decision expected by early 2026.
The positive opinion is based on results from two Phase 3 trials: ENERGIZE-T in transfusion-dependent patients and ENERGIZE in non-transfusion-dependent patients. The studies evaluated mitapivat 100 mg twice daily against placebo. According to InvestingPro analysis, Agios has demonstrated strong market momentum with a 50.11% price return over the past six months, suggesting investor confidence in the company’s clinical development programs. Discover more insights about AGIO and 1,400+ other stocks with InvestingPro’s comprehensive Research Reports.
Avanzanite, which entered an exclusive partnership with Agios (NASDAQ:AGIO) in June 2025 to commercialize PYRUKYND across Europe, the UK, and Switzerland, will manage the European distribution of the medication pending approval.
"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, in the statement. With a market capitalization of $2.41 billion and its next earnings report scheduled for October 30, 2025, investors are closely watching Agios’s commercial expansion efforts.
Thalassemia is a rare inherited blood disorder that disrupts hemoglobin production, reducing the number of circulating red blood cells and causing anemia, fatigue, and serious complications. Patients experience significant disease burden, including comorbidities, reduced quality of life, and shortened life expectancy.
In other recent news, Agios Pharmaceuticals reported a notable 45% increase in net revenue for the second quarter of 2025, reaching $12.5 million. This significant growth highlights the company’s strategic efforts to expand its product portfolio and prepare for potential new product launches, especially in the rare disease sector. Additionally, the European Medicines Agency’s Committee for Medicinal Products for Human Use has given a positive opinion on Agios’ treatment, PYRUKYND, for anemia associated with thalassemia. This recommendation applies to adult patients with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The final decision from the European Commission is anticipated by early 2026. These developments indicate Agios’ ongoing focus on addressing rare diseases and expanding its market presence.
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