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AGIO Stock Falls as FDA Delays Decision on Pyrukynd for Thalassemia

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Key Takeaways

  • AGIO stock dropped 11% as the FDA extended Pyrukynd's thalassemia review by three months.
  • The delay follows Agios' proposed REMS to address liver injury risks flagged in original filings.
  • A decision is now expected on Dec. 7, 2025, instead of the original September target date.

Shares of Agios Pharmaceuticals (AGIO - Free Report) fell 11% on Thursday after the company announced that the FDA had extended the review timeline for the supplemental new drug application (sNDA) seeking label expansion for its sole marketed drug, Pyrukynd (mitapivat), in the thalassemia indication by an additional three months.

A final decision from the regulatory body is now expected on Dec. 7, 2025.

The sNDA, which is supported by data from two separate phase III studies, ENERGIZE and ENERGIZE-T, is seeking approval of Pyrukynd, an oral pyruvate kinase (PK) activator, for the treatment of alpha or beta-thalassemia in adult patients who are non-transfusion-dependent (NTD) and transfusion-dependent (TD), respectively.

Agios recently responded to an FDA information request by submitting a proposed Risk Evaluation and Mitigation Strategy (REMS) to mitigate the risk of hepatocellular (liver) injury noted in the original sNDA for Pyrukynd. Since the REMS submission counts as a major amendment, the FDA has extended the sNDA review timeline by three months. The extension is not related to any new efficacy or safety data, as none were requested by the FDA or provided by the company.

AGIO's Price Performance

Year to date, shares of Agios have increased 10% compared with the industry’s rise of 4.1%.

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AGIO's Recent Development Activities With Pyrukynd

The FDA accepted AGIO’s sNDA seeking label expansion for Pyrukynd for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in January 2025.

A final decision from the FDA was previously expected on Sept. 7, 2025. A potential approval would have made Pyrukynd the first oral therapy for use in all thalassemia subtypes.

However, now that the FDA has extended the review time, the commercial launch of Pyrukynd in the thalassemia indication is likely to be delayed. The company previously expected to launch Pyrukynd in this indication before the end of 2025.

Last December, Agios revealed that two thalassemia patients who received Pyrukynd across the ENERGIZE and ENERGIZE-T studies reported liver injury in the first six months of treatment.

Pyrukynd is currently approved by the FDA to treat adults with PK deficiency, a rare and debilitating blood disorder. It is the sole marketed product in the company’s portfolio.

In the first half of 2025, Pyrukynd recorded sales of $21.2 million, reflecting a 26% increase year over year.

Agios is also evaluating Pyrukynd in two late-stage studies, ACTIVATE-Kids and ACTIVATE-KidsT, for treating PK deficiency in pediatric patients who are not regularly transfused and are regularly transfused, respectively.

AGIO's Zacks Rank & Stocks to Consider

Agios currently carries a Zacks Rank #4 (Sell).

Some better-ranked stocks in the biotech sector are Akero Therapeutics (AKRO - Free Report) , Allogene Therapeutics (ALLO - Free Report) and Adaptive Biotechnologies (ADPT - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Akero Therapeutics’ 2025 loss per share have narrowed from $3.93 to $3.85. Loss per share estimates for 2026 have narrowed from $4.27 to $4.14 during the same period. AKRO stock has surged 58.3% year to date.

Akero Therapeutics’ earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 49.24%.

In the past 60 days, estimates for Allogene Therapeutics’ 2025 loss per share have narrowed from $1.02 to 98 cents. Loss per share estimates for 2026 have narrowed from 97 cents to 90 cents during the same period. ALLO stock has plunged 47.9% year to date.

Allogene Therapeutics’ earnings beat estimates in three of the trailing four quarters, while meeting the same on the remaining occasion, the average surprise being 14.03%.

In the past 60 days, estimates for Adaptive Biotechnologies’ 2025 loss per share have narrowed from 87 cents to 71 cents. Loss per share estimates for 2026 have narrowed from 65 cents to 60 cents during the same period. ADPT stock has soared 117.1% year to date.

Adaptive Biotechnologies’ earnings beat estimates in each of the trailing four quarters, the average surprise being 23.99%.

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