AGIO Agios Pharm

The FDA has approved Agios Pharmaceuticals' AQVESME (mitapivat) for treating hemolytic anemia in adults with pyruvate kinase deficiency. This approval is based on two Phase 3 trials, ACTIVATE and ACTIVATE-T, which showed significant increases in hemoglobin and reductions in transfusion burden. AQVESME is the first and only approved therapy for this rare, lifelong genetic disease.

Agios Pharmaceuticals (NASDAQ:AGIO) shares fell 5.3% on Friday, trading at $27.6350 on lower than average volume after opening at $27.55. Despite meeting quarterly EPS estimates and showing a 43.3% year-over-year revenue increase, the company remains unprofitable with a negative net margin of approximately 896%. Analyst ratings for AGIO are varied, with a consensus "Moderate Buy" and a $38.89 price target, although some analysts have recently cut their price objectives significantly.

BofA Securities increased its price target for Agios Pharma (NASDAQ:AGIO) to $34.00 from $32.00, maintaining a Buy rating, following the FDA approval of its drug mitapivat (AQVESME). This drug is now the sole treatment approved for both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, leading BofA to raise its probability of success for thalassemia to 100%. The stock recently saw a 19.26% return, and the drug is anticipated to be available in late January 2026 with an expectation of $1 billion in peak sales.

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) saw an 18.63% increase in its stock after receiving FDA approval for Aqvesme, its anemia treatment for patients with alpha or beta thalassemia. The drug is the only FDA-approved medicine for both non-transfusion-dependent and transfusion-dependent cases. While effective, the approval includes a Risk Evaluation and Mitigation Strategy (REMS) due to potential hepatocellular injury, requiring liver monitoring and specific patient, physician, and pharmacist certifications.

Agios Pharmaceuticals (AGIO) received FDA approval for AQVESME (mitapivat) to treat anemia in adults with alpha- or beta-thalassemia, leading to a 14.6% stock increase. This first-in-class approval significantly de-risks the drug's near-term launch and transforms AQVESME into a new branded option in the thalassemia market. The company projects substantial revenue growth by 2028, with a fair value estimate suggesting a 10% upside to its current price, despite ongoing losses.

BofA has increased its price target for Agios Pharmaceuticals (AGIO) shares to $34 from $32, while maintaining a Buy rating. This adjustment follows FDA approval for mitapivat, making it the only drug approved for both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The firm anticipates no significant impact on uptake from the REMS and black box warning for hepatocellular injury.