Agios Awaits Pyrukynd Decision in Thalassemia as FDA Misses Due Date

Agios Pharmaceuticals AGIO announced that the FDA has not yet given its decision related to the company’s supplemental new drug application (sNDA) seeking approval for its only marketed drug, Pyrukynd (mitapivat), for treating thalassemia.

The sNDA sought approval for Pyrukynd for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. A final decision from the regulatory body was previously expected on Dec. 7, 2025.

With the FDA missing its target deadline, the sNDA for Pyrukynd in thalassemia remains under active review, and Agios is still awaiting clarity on the timing of the agency’s decision. The regulatory body in the United States has not provided a timeline for its regulatory decision. It has also not requested any new or additional efficacy or safety data.

An oral pyruvate kinase (PK) activator, Pyrukynd, is currently approved in the United States and Europe for treating hemolytic anemia in adults with PK deficiency, a rare and debilitating blood disorder.

AGIO Price Performance

Year to date, shares of Agios have declined 17% against the industry’s increase of 19.3%.

AGIO’s Recent Hurdles With Pyrukynd Label Expansion Studies

We remind investors that in September 2025, the FDA extended the review timeline for the sNDA seeking label expansion for Pyrukynd in the thalassemia indication by an additional three months.

The extension was due to the FDA’s request for a Risk Evaluation and Mitigation Strategy (REMS) to address the potential risk of hepatocellular (liver) injury mentioned in the original sNDA for Pyrukynd. The extension was not related to any new efficacy or safety data.

With the FDA missing the latest due date, a potential approval for Pyrukynd for the thalassemia indication is likely to be delayed further.

Recently, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending the marketing authorization application seeking label expansion for Pyrukynd in the thalassemia indication. A final decision from the European Commission is expected in early 2026.

Besides thalassemia, Pyrukynd is also being studied for treating sickle cell disease (SCD).

Last month, Agios reported mixed top-line data from the phase III RISE UP study, which evaluated Pyrukynd in patients aged 16 years or older with SCD.

While the study did meet its primary endpoint of improving patients’ hemoglobin levels, it failed to achieve the other co-primary endpoint of reducing the annualized rate of sickle cell pain crises. Shares of the company tanked on this news.

AGIO’s Zacks Rank & Stocks to Consider

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

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

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have increased from $7.28 to $7.54. EPS estimates for 2026 have moved up from $7.78 to $8.15 during the same period. ANIP stock has surged 47.2% year to date.

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

In the past 60 days, estimates for CorMedix’s 2025 EPS have increased from $1.83 to $2.87. EPS estimates for 2026 have moved up from $2.48 to $2.88 during the same period. CRMD stock has rallied 39% year to date.

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

In the past 60 days, estimates for Castle Biosciences’ loss per share have narrowed from 65 cents to 23 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from $2.10 to $1.42. Year to date, shares of CSTL have rallied 43%.

Castle Biosciences’ earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, the average surprise being 66.11%.

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This article originally published on Zacks Investment Research (zacks.com).

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