Agios: FDA Approval of Aqvesme in Thalassemia Unlocks Transformational Market Opportunity, Supporting Buy Rating and Higher Target

H.C. Wainwright analyst Emily Bodnar maintained a Buy rating on Agios Pharma today and set a price target of $62.00.

Emily Bodnar has given his Buy rating due to a combination of factors tied to the recent FDA approval of mitapivat (Aqvesme) for thalassemia and its commercial potential. She views the broad label in both non‑transfusion‑dependent and transfusion‑dependent alpha- and beta-thalassemia as a transformative milestone, positioning Aqvesme as the only approved oral therapy for a wide spectrum of anemic patients who previously had few or no options. The strong Phase 3 data in ENERGIZE and ENERGIZE‑T, which met primary and key secondary endpoints in a sizable patient population, underpins confidence in both efficacy and market uptake. In her assessment, the required REMS program and black box warning for hepatocellular injury are manageable, given the clear monitoring protocols, the reversibility of liver events upon drug discontinuation, and the fact that many thalassemia patients already undergo routine liver testing due to other hepatotoxic treatments.

Emily also believes that the REMS and safety labeling are unlikely to materially slow adoption, as specialist prescribers are already accustomed to similar risk‑management frameworks and survey work indicates limited impact on prescribing behavior. The company’s existing commercial platform from Pyrukynd, combined with a tailored dual-brand strategy that separates the PKD and thalassemia indications, is expected to support an efficient and effective launch. With product availability anticipated following REMS implementation, and early prescription interest already possible, she sees Agios as well positioned to translate this regulatory win into growing revenues. These dynamics, together with the strengthened risk‑benefit profile and expanded addressable market, support her decision to maintain a Buy rating and increase the price target.

In another report released today, Bank of America Securities also reiterated a Buy rating on the stock with a $34.00 price target.