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AGIO's Filing for Mitapivat in SCD Gets FDA's Priority Tag, Stock Up
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Key Takeaways
Agios received FDA priority review for mitapivat in sickle cell disease under accelerated approval.
AGIO expects an FDA decision by Nov. 1, 2026, after the agency accepted the application.
Mitapivat could become the first oral PK activator approved for sickle cell disease upon potential approval.
Agios Pharmaceuticals (AGIO - Free Report) announced that the FDA has accepted its supplemental new drug application (sNDA) seeking approval for its lead product, mitapivat, an oral pyruvate kinase (PK) activator, for the treatment of sickle cell disease (SCD).
The sNDA for mitapivat in SCD is submitted under the FDA’s accelerated approval pathway.
With the FDA granting a priority review to the sNDA, a decision from the regulatory body is expected on Nov 1, 2026.
A priority review designation means the FDA’s goal is to take action on an application within six months compared with 10 months under standard review.
If approved, mitapivat is likely to become the first oral PK activator to be approved for patients with SCD.
Shares of Agios were up 17.7% yesterday following the announcement of the news.
AGIO’s Price Performance
Year to date, shares of Agios have rallied 61.7% compared with the industry’s increase of 6.8%.
Image Source: Zacks Investment Research
Mitapivat is marketed under two brand names in the United States — Pyrukynd and Aqvesme. While Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with PK deficiency, Aqvesme was approved for the treatment of anemia in adults with alpha- or beta-thalassemia in December 2025. Aqvesme was launched in the United States following the implementation of the REMS program in January 2026.
Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications. While Pyrukynd was already approved in the European Union for PK deficiency, it secured approval for the thalassemia indication in May 2026.
Agios’ Recent Development With Mitapivat in SCD
In May 2026, Agios submitted the sNDA to the FDA seeking accelerated approval for mitapivat for the treatment of SCD.
The sNDA follows alignment with the FDA on a confirmatory study designed to support mitapivat’s accelerated approval for SCD. The study is designed to assess mitapivat’s ability to reduce transfusion burden in patients with SCD, with the primary endpoint of achieving transfusion-free status at week 52.
In November, AGIO reported top-line data from the phase III portion of the RISE UP study, which evaluated mitapivat in patients aged 16 years and older with SCD. Although the study did not meet its primary endpoints, the company’s findings showed clearer clinical benefits of the drug in patients.
During a meeting with the FDA, Agios presented the mixed RISE UP study results, following which the agency advised the company to submit a proposal for a confirmatory clinical study design for review to support accelerated approval.
If mitapivat is approved for SCD, a confirmatory study would be required to convert accelerated approval to traditional approval.
Over the past 60 days, estimates for Amarin’s 2026 loss per share have narrowed from $15.20 to 65 cents. Over the same period, loss per share estimates for 2027 have narrowed from $13.00 to 51 cents. AMRN shares have risen 10.8% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 50.02%.
Over the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2026 earnings per share have risen from $1.24 to $1.25, while estimates for 2027 have increased from $1.70 to $1.76 during the same time. KNSA shares have soared 63.1% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four quarters, while missing the same on the remaining two occasions, with the average surprise being 1.53%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $3.02, while estimates for 2027 have increased from $2.91 to $4.92 during the same time. LQDA shares have surged 135.5% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.
View the Zacks #1 Rank List
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AGIO's Filing for Mitapivat in SCD Gets FDA's Priority Tag, Stock Up
Key Takeaways
Agios Pharmaceuticals (AGIO - Free Report) announced that the FDA has accepted its supplemental new drug application (sNDA) seeking approval for its lead product, mitapivat, an oral pyruvate kinase (PK) activator, for the treatment of sickle cell disease (SCD).
The sNDA for mitapivat in SCD is submitted under the FDA’s accelerated approval pathway.
With the FDA granting a priority review to the sNDA, a decision from the regulatory body is expected on Nov 1, 2026.
A priority review designation means the FDA’s goal is to take action on an application within six months compared with 10 months under standard review.
If approved, mitapivat is likely to become the first oral PK activator to be approved for patients with SCD.
Shares of Agios were up 17.7% yesterday following the announcement of the news.
AGIO’s Price Performance
Year to date, shares of Agios have rallied 61.7% compared with the industry’s increase of 6.8%.
Image Source: Zacks Investment Research
Mitapivat is marketed under two brand names in the United States — Pyrukynd and Aqvesme. While Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with PK deficiency, Aqvesme was approved for the treatment of anemia in adults with alpha- or beta-thalassemia in December 2025. Aqvesme was launched in the United States following the implementation of the REMS program in January 2026.
Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications. While Pyrukynd was already approved in the European Union for PK deficiency, it secured approval for the thalassemia indication in May 2026.
Agios’ Recent Development With Mitapivat in SCD
In May 2026, Agios submitted the sNDA to the FDA seeking accelerated approval for mitapivat for the treatment of SCD.
The sNDA follows alignment with the FDA on a confirmatory study designed to support mitapivat’s accelerated approval for SCD. The study is designed to assess mitapivat’s ability to reduce transfusion burden in patients with SCD, with the primary endpoint of achieving transfusion-free status at week 52.
In November, AGIO reported top-line data from the phase III portion of the RISE UP study, which evaluated mitapivat in patients aged 16 years and older with SCD. Although the study did not meet its primary endpoints, the company’s findings showed clearer clinical benefits of the drug in patients.
During a meeting with the FDA, Agios presented the mixed RISE UP study results, following which the agency advised the company to submit a proposal for a confirmatory clinical study design for review to support accelerated approval.
If mitapivat is approved for SCD, a confirmatory study would be required to convert accelerated approval to traditional approval.
Agios Pharmaceuticals, Inc. Price
Agios Pharmaceuticals, Inc. price | Agios Pharmaceuticals, Inc. Quote
AGIO Zacks Rank & Stocks to Consider
Agios currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amarin (AMRN - Free Report) , Kiniksa Pharmaceuticals (KNSA - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting 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 Amarin’s 2026 loss per share have narrowed from $15.20 to 65 cents. Over the same period, loss per share estimates for 2027 have narrowed from $13.00 to 51 cents. AMRN shares have risen 10.8% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 50.02%.
Over the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2026 earnings per share have risen from $1.24 to $1.25, while estimates for 2027 have increased from $1.70 to $1.76 during the same time. KNSA shares have soared 63.1% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four quarters, while missing the same on the remaining two occasions, with the average surprise being 1.53%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $3.02, while estimates for 2027 have increased from $2.91 to $4.92 during the same time. LQDA shares have surged 135.5% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.