This section highlights FDA-related milestones and regulatory updates for drugs developed by Agios Pharmaceuticals (AGIO).
Over the past two years, Agios Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AG-946, Mitapivat, and Mitapivat. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
AG-946 - FDA Regulatory Timeline and Events
AG-946 is a drug developed by Agios Pharmaceuticals for the following indication: PKR activator, in healthy volunteers.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AG-946
- Announced Date:
- September 11, 2024
- Indication:
- PKR activator, in healthy volunteers
Announcement
Agios Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company's novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS).
AI Summary
Agios Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its novel pyruvate kinase (PK) activator tebapivat (AG-946) for treating myelodysplastic syndromes (MDS). This designation highlights the potential of tebapivat as a new oral therapy for patients with lower-risk MDS, who suffer from anemia due to ineffective erythropoiesis. It is estimated that around 75,000 to 80,000 patients in the U.S. and Europe could benefit from this treatment, which targets approximately 70% of MDS cases.
The designation provides Agios with important incentives, including tax credits, waivers for certain FDA fees, and the possibility of up to seven years of market exclusivity upon approval. Agios has already completed a Phase 2a study and is initiating a Phase 2b study to further evaluate tebapivat’s effectiveness and safety in this patient population.
Read Announcement
Mitapivat - FDA Regulatory Timeline and Events
Mitapivat is a drug developed by Agios Pharmaceuticals for the following indication: Sickle cell disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Mitapivat
- Announced Date:
- May 14, 2025
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals announced that new data on the company's PK activators, mitapivat and tebapivat, will be featured in oral and poster presentations during the 30th European Hematology Association (EHA) Congress (EHA 2025) in Milan, Italy, June 12-15, 2025.
AI Summary
Agios Pharmaceuticals announced that new data on its pyruvate kinase (PK) activators, mitapivat and tebapivat, will be featured at the 30th European Hematology Association (EHA) Congress in Milan, Italy, from June 12-15, 2025. The company will present both oral and poster sessions showcasing robust clinical and preclinical results. Key highlights include data from the ACTIVATE-KidsT Phase 3 trial, which evaluated mitapivat in children with PK deficiency and demonstrated a reduction in transfusion needs, and long-term safety and efficacy findings in patients with sickle cell disease. Preclinical studies will also be discussed, including data showing that tebapivat reduces red blood cell sickling and adhesion. These presentations underscore the therapeutic potential of PK activation for rare blood disorders, offering promising treatment options for conditions such as PK deficiency, sickle cell disease, and myelodysplastic syndromes.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- February 13, 2025
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals, announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused achieved its primary endpoint of hemoglobin response.
AI Summary
Agios Pharmaceuticals announced encouraging results from its ACTIVATE-Kids Phase 3 study evaluating mitapivat in children aged 1 to under 18 years with pyruvate kinase (PK) deficiency who are not regularly transfused. The study met its primary endpoint, showing a significant hemoglobin response, with treated patients achieving a sustained increase in hemoglobin levels compared to those on placebo. The trial’s design included a 20‑week double-blind period, and the safety results in children were consistent with what had been observed in adult patients with PK deficiency.
These findings highlight mitapivat’s potential to improve anemia and related symptoms in pediatric patients with this rare hemolytic anemia. The positive outcome is seen as an important milestone in advancing treatment options for children affected by PK deficiency.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- January 8, 2025
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) accepted the company's supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
AI Summary
Agios Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat). The application targets adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, a rare inherited blood disorder that causes chronic anemia and can lead to serious health complications. The FDA will conduct a standard review, with a decision expected by September 7, 2025.
This important submission is part of Agios’ commitment to developing innovative treatments for rare diseases. PYRUKYND is an oral, disease-modifying medication that may provide a much-needed treatment option for thalassemia patients with varying transfusion needs. The company looks forward to working closely with the FDA as the review process continues, which could help improve the quality of life for many patients suffering from this debilitating condition.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- December 18, 2024
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals announced that the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease.
AI Summary
Agios Pharmaceuticals announced that the European Commission has given a positive decision for the orphan medicinal product designation of mitapivat as a treatment for sickle cell disease. Mitapivat, an oral small molecule that activates pyruvate kinase, is part of Agios’s efforts to develop novel therapies addressing serious, rare conditions. The orphan designation supports further development by offering benefits like reduced fees and a 10-year period of market exclusivity.
The decision by the European Commission highlights the urgent need for new treatment options for patients with sickle cell disease. Agios, known for its pioneering work in cellular metabolism and PK activation, is currently running the fully enrolled Phase 3 RISE UP study, with results expected to be shared in late 2025.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- December 8, 2024
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals, today presented positive results from the Phase 3 ENERGIZE-T study investigating mitapivat, an oral, small molecule PK activator, in adults with transfusion-dependent alpha- or beta-thalassemia.
AI Summary
Agios Pharmaceuticals presented strong Phase 3 data from its ENERGIZE-T study, which tested mitapivat, an oral small molecule pyruvate kinase (PK) activator, in adults with transfusion-dependent alpha- or beta-thalassemia. The study met its primary endpoint by showing a significant reduction in the number of red blood cell transfusions compared to placebo. In addition to the primary outcome, all key secondary endpoints were achieved, with patients on mitapivat experiencing marked improvements in transfusion reduction response over different time frames during the 48-week trial. These results suggest that mitapivat could offer a disease-modifying option for patients suffering from thalassemia, a condition with limited current treatment choices. The study’s success positions mitapivat as a promising treatment candidate that may transform care for patients with this debilitating blood disorder.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- August 1, 2024
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals, announced topline results from the global Phase 3 ACTIVATE-KidsT study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused.
AI Summary
Agios Pharmaceuticals announced topline results from its global Phase 3 ACTIVATE-KidsT study of mitapivat in children aged 1 to less than 18 years with pyruvate kinase (PK) deficiency who require regular blood transfusions. Although the study did not meet the prespecified statistical criterion for the primary endpoint using Bayesian methods, the clinical results were promising. In the study, 28.1% of patients receiving mitapivat achieved a reduction in transfusion needs compared to 11.8% of patients given a placebo. Furthermore, only the mitapivat group showed a transfusion-free response and normalization of hemoglobin levels. The safety profile in children was consistent with what has been seen in adults, with mitapivat being generally safe and well-tolerated over the 32-week period.
These encouraging findings mark Agios' first pediatric data readout for mitapivat and support its potential as a disease-modifying therapy for children with PK deficiency.
Read Announcement- Drug:
- Mitapivat
- Announced Date:
- June 15, 2024
- Indication:
- Sickle cell disease
Announcement
Agios Pharmaceuticals, today presented detailed results from the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia in a plenary session (abstract #S104) at the European Hematology Association 2024 (EHA2024) Hybrid Congress, which is being held June 13-16, 2024, in Madrid, Spain.
AI Summary
Agios Pharmaceuticals presented detailed results from the global Phase 3 ENERGIZE study at the European Hematology Association 2024 Hybrid Congress in Madrid, Spain. The study evaluated the efficacy of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia and showed a statistically significant increase in hemoglobin response compared to placebo. In addition, accompanying poster data highlighted meaningful improvements in fatigue, exercise capacity, and overall quality of life for patients treated with mitapivat. The study achieved its primary endpoint and demonstrated improvements in key secondary endpoints including changes in FACIT-Fatigue scores and hemoglobin levels. These findings suggest that mitapivat could become an important oral treatment option for NTD thalassemia, offering a convenient pill-based therapy for patients managing the disease’s symptoms and its impact on daily activities.
Read Announcement
Mitapivat (ACTIVATE) - FDA Regulatory Timeline and Events
Mitapivat (ACTIVATE) is a drug developed by Agios Pharmaceuticals for the following indication: PK deficiency who do not receive regular transfusions.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Mitapivat (ACTIVATE)
- Announced Date:
- June 3, 2024
- Indication:
- PK deficiency who do not receive regular transfusions
Announcement
Agios Pharmaceuticals, announced that the global Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent (TD) alpha- or beta-thalassemia achieved its primary endpoint of transfusion reduction response. Statistical significance was also achieved for all key secondary endpoints evaluating additional measures of reduction of transfusion burden compared to placebo.
AI Summary
Agios Pharmaceuticals announced that its global Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent (TD) alpha- and beta-thalassemia achieved its primary endpoint of reducing transfusion burdens. The study demonstrated that patients treated with mitapivat experienced a statistically significant reduction in red blood cell transfusions compared to those receiving a placebo. In addition, all key secondary endpoints, which measured further reductions in transfusion needs, also met statistical significance. These results indicate that mitapivat’s unique mechanism may improve red blood cell health and offer a beneficial oral treatment option for thalassemia patients, potentially lowering risks associated with repeated transfusions such as iron overload and immune reactions. Based on these positive findings, Agios plans to submit a U.S. marketing application for mitapivat by the end of 2024 to help transform care for patients with transfusion-dependent thalassemia.
Read Announcement
