AGIO's Mitapivat Gets FDA Nod for Thalassemia Indication with REMS in Label

Agios Pharmaceuticals AGIO announced that the FDA has approved Aqvesme (mitapivat) for the treatment of adult patients with alpha- or beta-thalassemia. With this approval, Aqvesme becomes the only FDA-approved drug for the treatment of non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

Mitapivat is already approved by the name of Pyrukynd to treat hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency, a rare and debilitating blood disorder.

For the thalassemia indication, Agios will market mitapivat under the brand name Aqvesme in the United States. Aqvesme is expected to be launched in late January 2026, following implementation of the AQVESME REMS program. Mitapivat will continue to be marketed as Pyrukynd for its PK deficiency indication in the United States.

Aqvesme’s Approval Based on AGIO’s ENERGIZE and ENERGIZE-T Studies

The FDA approval of Aqvesme is supported by data from phase III ENERGIZE and ENERGIZE-T studies, which demonstrated that Aqvesme led to statistically significant and clinically meaningful improvements in hemoglobin and fatigue levels as well as reduction in blood transfusion requirements compared with placebo in non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia patients.

A small portion of the patient population treated with Aqvesme experienced hepatocellular (liver) injury, including cases requiring hospitalization. To address the risk, the FDA has mandated AQVESME REMS, a Risk Evaluation and Mitigation Strategy program. Under this program, patients must have liver tests before the first dose and at regular intervals thereafter.

The inclusion of REMS in Aqvesme’s label likely disappointed investors, causing the stock to decline around 1.5% on Tuesday.

Over the past year, AGIO’s shares have declined 28.1% while the industry has risen 16.8%.

The FDA was originally scheduled to issue its regulatory decision on the supplemental new drug application (sNDA) seeking expanded use of Pyrukynd (mitapivat), for the treatment of thalassemia by September 2025. However, the FDA extended the review period by three months and was expected to deliver the decision by Dec.7, 2025. The regulatory authority ultimately announced its decision on Dec.23, 2025, to facilitate the implementation of the REMS program.

Due to the inclusion of the AQVESME REMS program, mitapivat will be commercialized under a different brand name in the United States for the thalassemia indication.

Agios has also filed a regulatory application seeking approval of Aqvesme in the European Union. In October, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending approval of the marketing authorization application to expand Pyrukynd’s label for the thalassemia indication. A final decision from the European Commission is expected in early 2026.

In markets outside the United States, mitapivat will continue to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.

Besides thalassemia, Pyrukynd is also being studied for sickle cell disease (“SCD”). Agios plans to hold a pre-supplemental new drug application meeting with the FDA in the first quarter of 2026, following which it will file for approval of Pyrukynd for the SCD indication.

AGIO’s Zacks Rank & Stocks to Consider

AGIO currently carries a Zacks Rank #3 (Hold).

Some top-ranked stocks in the biotech sector are ANI Pharmaceuticals ANIP, CorMedix CRMD, and Castle Biosciences CSTL. ANIP, CRMD, and CSTL currently sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for ANI Pharmaceuticals’ 2025 adjusted EPS have increased from $7.29 to $7.56. Over the same period, EPS estimates for 2026 have surged from $7.81 to $8.08. Shares of ANIP have surged 49.1% in the past year.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 21.24%.

Over the past 60 days, estimates for CorMedix’s 2025 EPS have increased from $1.85 to $2.87, while 2026 EPS estimates have risen from $2.49 to $2.88 over the same period. Shares of CRMD have surged 43.8% over the past year.

CorMedix’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 27.04%.

Over the past 60 days, the loss estimate for Castle Biosciences has narrowed from 64 cents to 34 cents in 2025. Over the same period, loss estimates for 2026 have improved from $1.82 to $1.06. CSTL stock has rallied 45% over the past year.

Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining quarter, with the average surprise being 66.11%.

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