Agios Submits sNDA for Accelerated Approval of Mitapivat in Sickle Cell Disease
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 12 2026
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Source: Newsfilter
- Clinical Trial Design: Agios' supplemental New Drug Application (sNDA) submission is based on an agreement with the FDA requiring a confirmatory clinical trial to demonstrate the clinical benefit of mitapivat in reducing transfusion burden in sickle cell disease, with approximately 159 patients expected to be enrolled over a 52-week period.
- Key Endpoint Setting: The primary endpoint of the confirmatory trial is the transfusion-free rate from Week 4 to Week 52, aiming to showcase mitapivat's effectiveness in lowering transfusion needs compared to placebo, reflecting its potential market value in sickle cell disease treatment.
- Clinical Data Support: In the RISE UP Phase 3 trial, mitapivat demonstrated significant improvements in hemoglobin concentration and reduced hemolysis, translating clinically into meaningful reductions in pain crises and hospitalizations, indicating its strong potential in treating sickle cell disease.
- Market Outlook: Agios expects to receive notice of the sNDA filing acceptance from the FDA in the third quarter of 2026, and if approved, mitapivat will become the first PK activator approved in the U.S., addressing the urgent need for new therapies among sickle cell disease patients.
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Analyst Views on AGIO
Wall Street analysts forecast AGIO stock price to rise
9 Analyst Rating
6 Buy
3 Hold
0 Sell
Moderate Buy
Current: 30.420
Low
25.00
Averages
36.14
High
62.00
Current: 30.420
Low
25.00
Averages
36.14
High
62.00
About AGIO
Agios Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on developing and delivering transformative therapies for patients living with rare diseases. It markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for debilitating hemolytic anemia. Its lead product candidate in its portfolio, PYRUKYND (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. It is also developing tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes, or LR MDS, and hemolytic anemias; AG-181, its phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU; and AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., targeting the transmembrane serine protease 6, or TMPRSS6 gene for the potential treatment of polycythemia vera, or PV.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Agios Halts Development of LR-MDS Drug Tebapivat
- Clinical Trial Results: Agios announced that its drug tebapivat for lower-risk myelodysplastic syndromes (LR-MDS) did not meet the predefined clinical advancement threshold in the Phase 2b trial, which, while showing biological activity, failed to demonstrate clinical benefit in a sufficient proportion of patients, leading to the decision to halt further development.
- Patient Population Characteristics: The trial enrolled 65 patients with LR-MDS, evaluating different doses (10mg, 15mg, 20mg) of tebapivat in a heavily pretreated and heterogeneous population, highlighting the biological complexity of the disease and the challenges in identifying patients likely to benefit.
- Safety Assessment: Although tebapivat was well tolerated across all dose levels with no new safety signals identified, the lack of observed clinical benefit necessitated Agios's decision to discontinue further development in LR-MDS.
- Future Outlook: Agios remains optimistic about tebapivat's potential in sickle cell disease, with topline data from the Phase 2 trial expected in the second half of 2026, continuing to explore its applications in other rare hematologic diseases.
See More
Regulatory Approvals and Collaborations in Biotech Sector
- FDA Approval for AZN: AstraZeneca (AZN) secured FDA approval for the IMFINZI + BCG combination in high-risk non-muscle-invasive bladder cancer, supported by Phase 3 POTOMAC data showing a 32% reduction in recurrence or death, enhancing its competitive edge in oncology.
- JNJ TREMFYA Label Expansion: Johnson & Johnson (JNJ) received FDA approval to add structural joint-damage inhibition data to the TREMFYA label for active psoriatic arthritis, confirming its unique position in first-line treatment with no new safety signals, reinforcing its market leadership.
- LINZESS Approved for Children: Ironwood Pharmaceuticals (IRWD) gained FDA approval for LINZESS to treat functional constipation in children aged 2-5, based on a 12-week Phase 3 trial showing significant improvement in spontaneous bowel movements, expanding its pediatric market influence.
- AbbVie's DECNUPAZ Approval: AbbVie (ABBV) received FDA approval for DECNUPAZ to treat ultra-rare blood cancer BPDCN, supported by Phase 1 data showing a 69.7% composite complete response rate and a median duration of 9.7 months, marking a significant innovation in hematologic oncology.
See More
Agios Pharmaceuticals Halts Blood Cancer Treatment Trials
- Study Discontinuation: Agios Pharmaceuticals announced the discontinuation of studies for a treatment targeting myelodysplastic syndromes, resulting in a ~6% drop in premarket trading, indicating a strategic shift in the company's focus.
- Disappointing Trial Results: The 24-week dose-finding trial did not meet the predefined target for transfusion independence, reflecting insufficient efficacy of the treatment and impacting the company's future research direction.
- Safety Profile Maintained: Despite the unsatisfactory trial outcomes, Agios reported that the oral pyruvate kinase activator was well-tolerated across multiple dose levels with no new safety signals, suggesting potential for use in other indications.
- Future Research Plans: Agios is conducting another Phase 2 trial for sickle cell disease, with topline data expected in H2 2026, demonstrating the company's ongoing commitment to exploring alternative treatment avenues for growth.
See More
Agios Receives EU Approval for PYRUKYND
- Drug Approval: Agios Pharmaceuticals and its partner Avanzanite Bioscience announced that the European Commission has granted marketing authorization for their lead product PYRUKYND to treat transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking the second indication for the drug in the EU.
- Clinical Trial Support: The approval is based on a positive opinion from the European Medicines Agency (EMA) and relies on results from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated the efficacy and safety of Mitapivat in adults with thalassemia.
- Market Launch Plan: Under an exclusive agreement with Agios, Avanzanite Bioscience will commercialize and distribute PYRUKYND across the European Economic Area, the UK, and Switzerland, with CEO Adam Plich stating their commitment to collaborate with local authorities to drive a successful launch and ensure broad access.
- Stock Price Dynamics: AGIO's stock has traded between $22.24 and $46.00 over the past year, closing at $28.14, down 2.26%, and falling to $27.60 in after-hours trading, indicating a cautious market reaction to the new drug approval.
See More
Avanzanite Launches PYRUKYND in the EU
- Drug Approval: Avanzanite, in partnership with Agios, has received marketing authorization for PYRUKYND in the EU, introducing a new oral treatment option for adult patients with transfusion-dependent and non-transfusion-dependent alpha or beta-thalassaemia, marking its second indication in the EU.
- Clinical Trial Support: This approval is based on results from the global randomized double-blind ENERGIZE and ENERGIZE-T Phase 3 trials, demonstrating significant efficacy in improving patient quality of life and reducing transfusion burden, highlighting its clinical importance.
- Market Access Commitment: Avanzanite is committed to collaborating with local health authorities to ensure broad access to PYRUKYND across the EU, aiming to help all thalassaemia patients access this new therapy, reflecting the company's dedication to patient care.
- Commercial Platform Advantage: This launch marks Avanzanite's fourth drug introduction in the rare disease sector, showcasing its strong commercial platform across 32 European countries and its capability to partner with biotech innovators to enhance treatment accessibility for rare diseases.
See More
PYRUKYND Approved in EU for Thalassemia Treatment
- Regulatory Milestone: PYRUKYND (mitapivat) has received marketing authorization from the European Commission, becoming the only approved treatment for both transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking a significant advancement for thousands of patients.
- Clinical Trial Validation: This approval is based on results from global randomized Phase 3 trials, demonstrating PYRUKYND's efficacy in alleviating anemia symptoms and improving quality of life, highlighting its substantial clinical significance.
- Market Expansion Strategy: Agios has entered into an exclusive agreement with Avanzanite Bioscience for the commercialization and distribution of PYRUKYND across the European Economic Area, further solidifying its position in the global rare disease market.
- Global Recognition: With approvals in the U.S., Saudi Arabia, UAE, and now the EU, PYRUKYND reflects widespread demand and potential market opportunities globally, indicating promising future sales growth.
See More
Agios Halts Development of LR-MDS Drug Tebapivat
- Clinical Trial Results: Agios announced that its drug tebapivat for lower-risk myelodysplastic syndromes (LR-MDS) did not meet the predefined clinical advancement threshold in the Phase 2b trial, which, while showing biological activity, failed to demonstrate clinical benefit in a sufficient proportion of patients, leading to the decision to halt further development.
- Patient Population Characteristics: The trial enrolled 65 patients with LR-MDS, evaluating different doses (10mg, 15mg, 20mg) of tebapivat in a heavily pretreated and heterogeneous population, highlighting the biological complexity of the disease and the challenges in identifying patients likely to benefit.
- Safety Assessment: Although tebapivat was well tolerated across all dose levels with no new safety signals identified, the lack of observed clinical benefit necessitated Agios's decision to discontinue further development in LR-MDS.
- Future Outlook: Agios remains optimistic about tebapivat's potential in sickle cell disease, with topline data from the Phase 2 trial expected in the second half of 2026, continuing to explore its applications in other rare hematologic diseases.
See More
Regulatory Approvals and Collaborations in Biotech Sector
- FDA Approval for AZN: AstraZeneca (AZN) secured FDA approval for the IMFINZI + BCG combination in high-risk non-muscle-invasive bladder cancer, supported by Phase 3 POTOMAC data showing a 32% reduction in recurrence or death, enhancing its competitive edge in oncology.
- JNJ TREMFYA Label Expansion: Johnson & Johnson (JNJ) received FDA approval to add structural joint-damage inhibition data to the TREMFYA label for active psoriatic arthritis, confirming its unique position in first-line treatment with no new safety signals, reinforcing its market leadership.
- LINZESS Approved for Children: Ironwood Pharmaceuticals (IRWD) gained FDA approval for LINZESS to treat functional constipation in children aged 2-5, based on a 12-week Phase 3 trial showing significant improvement in spontaneous bowel movements, expanding its pediatric market influence.
- AbbVie's DECNUPAZ Approval: AbbVie (ABBV) received FDA approval for DECNUPAZ to treat ultra-rare blood cancer BPDCN, supported by Phase 1 data showing a 69.7% composite complete response rate and a median duration of 9.7 months, marking a significant innovation in hematologic oncology.
See More
Agios Pharmaceuticals Halts Blood Cancer Treatment Trials
- Study Discontinuation: Agios Pharmaceuticals announced the discontinuation of studies for a treatment targeting myelodysplastic syndromes, resulting in a ~6% drop in premarket trading, indicating a strategic shift in the company's focus.
- Disappointing Trial Results: The 24-week dose-finding trial did not meet the predefined target for transfusion independence, reflecting insufficient efficacy of the treatment and impacting the company's future research direction.
- Safety Profile Maintained: Despite the unsatisfactory trial outcomes, Agios reported that the oral pyruvate kinase activator was well-tolerated across multiple dose levels with no new safety signals, suggesting potential for use in other indications.
- Future Research Plans: Agios is conducting another Phase 2 trial for sickle cell disease, with topline data expected in H2 2026, demonstrating the company's ongoing commitment to exploring alternative treatment avenues for growth.
See More
Agios Receives EU Approval for PYRUKYND
- Drug Approval: Agios Pharmaceuticals and its partner Avanzanite Bioscience announced that the European Commission has granted marketing authorization for their lead product PYRUKYND to treat transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking the second indication for the drug in the EU.
- Clinical Trial Support: The approval is based on a positive opinion from the European Medicines Agency (EMA) and relies on results from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated the efficacy and safety of Mitapivat in adults with thalassemia.
- Market Launch Plan: Under an exclusive agreement with Agios, Avanzanite Bioscience will commercialize and distribute PYRUKYND across the European Economic Area, the UK, and Switzerland, with CEO Adam Plich stating their commitment to collaborate with local authorities to drive a successful launch and ensure broad access.
- Stock Price Dynamics: AGIO's stock has traded between $22.24 and $46.00 over the past year, closing at $28.14, down 2.26%, and falling to $27.60 in after-hours trading, indicating a cautious market reaction to the new drug approval.
See More
Avanzanite Launches PYRUKYND in the EU
- Drug Approval: Avanzanite, in partnership with Agios, has received marketing authorization for PYRUKYND in the EU, introducing a new oral treatment option for adult patients with transfusion-dependent and non-transfusion-dependent alpha or beta-thalassaemia, marking its second indication in the EU.
- Clinical Trial Support: This approval is based on results from the global randomized double-blind ENERGIZE and ENERGIZE-T Phase 3 trials, demonstrating significant efficacy in improving patient quality of life and reducing transfusion burden, highlighting its clinical importance.
- Market Access Commitment: Avanzanite is committed to collaborating with local health authorities to ensure broad access to PYRUKYND across the EU, aiming to help all thalassaemia patients access this new therapy, reflecting the company's dedication to patient care.
- Commercial Platform Advantage: This launch marks Avanzanite's fourth drug introduction in the rare disease sector, showcasing its strong commercial platform across 32 European countries and its capability to partner with biotech innovators to enhance treatment accessibility for rare diseases.
See More
PYRUKYND Approved in EU for Thalassemia Treatment
- Regulatory Milestone: PYRUKYND (mitapivat) has received marketing authorization from the European Commission, becoming the only approved treatment for both transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking a significant advancement for thousands of patients.
- Clinical Trial Validation: This approval is based on results from global randomized Phase 3 trials, demonstrating PYRUKYND's efficacy in alleviating anemia symptoms and improving quality of life, highlighting its substantial clinical significance.
- Market Expansion Strategy: Agios has entered into an exclusive agreement with Avanzanite Bioscience for the commercialization and distribution of PYRUKYND across the European Economic Area, further solidifying its position in the global rare disease market.
- Global Recognition: With approvals in the U.S., Saudi Arabia, UAE, and now the EU, PYRUKYND reflects widespread demand and potential market opportunities globally, indicating promising future sales growth.
See More
