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AGIO Rises on Mitapivat Progress Toward Accelerated Approval in SCD
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Key Takeaways
AGIO stock rises 14% after the company plans to seek accelerated approval for mitapivat in SCD.
AGIO shared RISE UP data with the FDA, which advised a confirmatory study to support approval.
AGIO submitted a confirmatory study design and plans an sNDA filing in the coming months.
Shares of Agios Pharmaceuticals (AGIO - Free Report) rallied 14% on Tuesday after the company announced its intention to seek accelerated approval for its lead drug, mitapivat, for the treatment of sickle cell disease (SCD), following a pre-supplemental new drug application (sNDA) meeting with the FDA.
Mitapivat is already marketed under two brand names — Pyrukynd and Aqvesme. Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency. Aqvesme is approved to treat anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.
Year to date, the stock has gained 24.3% compared with the industry’s 1.2% growth.
Image Source: Zacks Investment Research
Regulatory Feedback & Clinical Data Review
In November 2025, AGIO reported top-line data from the phase III RISE UP study of mitapivat for the treatment of patients with SCD. The study met the primary endpoint of hemoglobin response and demonstrated a reduction in the annualized rate of sickle cell pain crises compared with placebo, but failed to achieve statistical significance. The study also met key secondary endpoints of significant improvements in hemoglobin concentration and indirect bilirubin levels from baseline.
During the meeting with the FDA, the company presented data from its RISE UP clinical program, encompassing both phase II and phase III studies. Based on the discussion, the FDA advised Agios Pharmaceuticals to submit a proposal for a confirmatory clinical study to support accelerated approval.
The accelerated approval pathway is designed to expedite access to therapies addressing serious conditions, contingent upon subsequent validation through confirmatory studies.
AGIO Advances Confirmatory Study & sNDA Plans for Mitapivat in SCD
Agios Pharmaceuticals submitted its proposed confirmatory clinical study design to the FDA for review. The proposal incorporates a primary endpoint, distinct from those used in the RISE UP program and has been developed based on insights from analyses of RISE UP data, along with feedback from the FDA.
The company plans to submit the sNDA for mitapivat in SCD in the coming months and is closely engaged with the FDA to align on the requirements for the confirmatory study needed to support accelerated approval.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have declined from $2.55 to $2.51. CPRX shares have gained 1.2% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.89 to $3.08. INDV shares have surged 220.2% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $8.99 for 2026. Over the past year, shares of ANIP have rallied 10.9%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
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AGIO Rises on Mitapivat Progress Toward Accelerated Approval in SCD
Key Takeaways
Shares of Agios Pharmaceuticals (AGIO - Free Report) rallied 14% on Tuesday after the company announced its intention to seek accelerated approval for its lead drug, mitapivat, for the treatment of sickle cell disease (SCD), following a pre-supplemental new drug application (sNDA) meeting with the FDA.
Mitapivat is already marketed under two brand names — Pyrukynd and Aqvesme. Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency. Aqvesme is approved to treat anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.
Year to date, the stock has gained 24.3% compared with the industry’s 1.2% growth.
Image Source: Zacks Investment Research
Regulatory Feedback & Clinical Data Review
In November 2025, AGIO reported top-line data from the phase III RISE UP study of mitapivat for the treatment of patients with SCD. The study met the primary endpoint of hemoglobin response and demonstrated a reduction in the annualized rate of sickle cell pain crises compared with placebo, but failed to achieve statistical significance. The study also met key secondary endpoints of significant improvements in hemoglobin concentration and indirect bilirubin levels from baseline.
During the meeting with the FDA, the company presented data from its RISE UP clinical program, encompassing both phase II and phase III studies. Based on the discussion, the FDA advised Agios Pharmaceuticals to submit a proposal for a confirmatory clinical study to support accelerated approval.
The accelerated approval pathway is designed to expedite access to therapies addressing serious conditions, contingent upon subsequent validation through confirmatory studies.
AGIO Advances Confirmatory Study & sNDA Plans for Mitapivat in SCD
Agios Pharmaceuticals submitted its proposed confirmatory clinical study design to the FDA for review. The proposal incorporates a primary endpoint, distinct from those used in the RISE UP program and has been developed based on insights from analyses of RISE UP data, along with feedback from the FDA.
The company plans to submit the sNDA for mitapivat in SCD in the coming months and is closely engaged with the FDA to align on the requirements for the confirmatory study needed to support accelerated approval.
AGIO’s Zacks Rank & Stocks to Consider
AGIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals (CPRX - Free Report) and Indivior Pharmaceuticals (INDV - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy), and ANI Pharmaceuticals (ANIP - Free Report) , which presently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have declined from $2.55 to $2.51. CPRX shares have gained 1.2% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.89 to $3.08. INDV shares have surged 220.2% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $8.99 for 2026. Over the past year, shares of ANIP have rallied 10.9%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.