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Agios Seeks FDA Nod to Expand Mitapivat Label in Sickle Cell Disease
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Key Takeaways
Agios submitted an sNDA seeking accelerated approval of mitapivat for sickle cell disease.
AGIO aligned with the FDA on a confirmatory study to assess transfusion-free status at week 52.
Mitapivat showed clinical benefits in SCD patients despite missing primary endpoints in RISE UP.
Agios Pharmaceuticals (AGIO - Free Report) announced that it has submitted a supplemental new drug application (sNDA) to the FDA seeking accelerated approval for the label expansion of its lead drug, mitapivat, for the treatment of sickle cell disease (SCD). Agios expects to receive notice of the sNDA filing acceptance and anticipated review timeline in the third quarter of 2026, following the authority’s 60-day filing review period.
Mitapivat, an oral pyruvate kinase (PK) activator, 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 is approved for the treatment of 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, AGIO stock has risen 4.5% against the industry’s 0.5% decline.
Image Source: Zacks Investment Research
Confirmatory Study to Support Accelerated Approval for SCD
The sNDA follows alignment with the FDA on a confirmatory study designed to support mitapivat’s accelerated approval for SCD. The study will 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 the drug 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 $7.01 to $6.36. Over the same period, loss per share estimates for 2027 have narrowed from $5.50 to $4.64. AMRN shares have risen 7.1% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 50.02%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.03 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.46. INDV shares have risen 6.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.14 from $1.75. Over the same period, EPS estimates for 2027 have risen to $3.79 from $2.91. LQDA shares have gained 64.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
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Agios Seeks FDA Nod to Expand Mitapivat Label in Sickle Cell Disease
Key Takeaways
Agios Pharmaceuticals (AGIO - Free Report) announced that it has submitted a supplemental new drug application (sNDA) to the FDA seeking accelerated approval for the label expansion of its lead drug, mitapivat, for the treatment of sickle cell disease (SCD). Agios expects to receive notice of the sNDA filing acceptance and anticipated review timeline in the third quarter of 2026, following the authority’s 60-day filing review period.
Mitapivat, an oral pyruvate kinase (PK) activator, 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 is approved for the treatment of 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, AGIO stock has risen 4.5% against the industry’s 0.5% decline.
Image Source: Zacks Investment Research
Confirmatory Study to Support Accelerated Approval for SCD
The sNDA follows alignment with the FDA on a confirmatory study designed to support mitapivat’s accelerated approval for SCD. The study will 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 the drug 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.
AGIO’s Zacks Rank & Stocks to Consider
AGIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amarin Corporation (AMRN - Free Report) , Indivior Pharmaceuticals (INDV - 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 $7.01 to $6.36. Over the same period, loss per share estimates for 2027 have narrowed from $5.50 to $4.64. AMRN shares have risen 7.1% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 50.02%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.03 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.46. INDV shares have risen 6.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.14 from $1.75. Over the same period, EPS estimates for 2027 have risen to $3.79 from $2.91. LQDA shares have gained 64.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.