Agios Pharmaceuticals AGIO announced that its lead drug, Pyrukynd (mitapivat), an oral pyruvate kinase (PK) activator, has gained approval for a new indication in the European Union.
The European Commission (EC) approved Pyrukynd for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in adult patients. The approval was expected, as the Committee for Medicinal Products for Human Use of the European Medicines Agency had issued a positive opinion in October 2025 recommending Pyrukynd’s approval.
Agios’ partner, Avanzanite Bioscience B.V. will commercialize and distribute Pyrukynd across the European Economic Area, the United Kingdom and Switzerland.
With this approval, Pyrukynd becomes the only therapy authorized in the European Union for this broad thalassemia patient population.
Year to date, AGIO shares have risen 3.4% against the industry’s 0.3% decline.
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The FDA approved mitapivat under the brand name ‘Aqvesme’ for the treatment of adult patients with alpha- or beta-thalassemia in December 2025. Aqvesme was launched in the United States following the implementation of the REMS program in January 2026. AGIO continues to market the drug as Pyrukynd for thalassemia outside the United States (in Saudi Arabia and the United Arab Emirates).
Pyrukynd is also approved for the treatment of hemolytic anemia in adults with PK deficiency in the United States and Europe.
Pyrukynd’s EU Approval Based on AGIO’s Phase III Studies
The EC approval is supported by positive data from the phase III ENERGIZE and ENERGIZE-T studies, which demonstrated that Pyrukynd led to statistically significant and clinically meaningful improvements in hemoglobin levels and fatigue in patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The drug also reduced blood transfusion requirements compared with placebo.
More on AGIO’s Mitapivat
Besides approved indications, mitapivat is under regulatory review in the United States for the treatment of patients with sickle cell disease (SCD). Earlier this month, Agios submitted a supplemental new drug application to the FDA seeking accelerated approval for the potential label expansion of mitapivat in SCD.
The company expects to receive confirmation of the filing’s acceptance and the anticipated review timeline in the third quarter of 2026, following the authority’s 60-day filing review process.
AGIO’s Zacks Rank & Stocks to Consider
Agios currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals INDV and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy) and Immunocore IMCR, which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.10 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.47. INDV shares have risen 4.6% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 79.8% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents per share. IMCR shares have lost 16.9% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.
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This article originally published on Zacks Investment Research (zacks.com).