Agios Pharmaceuticals
Agios Pharmaceuticals works on 6 rare diseases tracked on Trial Friend, including Myelodysplastic Syndromes, Phenylketonuria, Polycythemia Vera and 3 more, with 13 active clinical trials (4 recruiting) and 2 FDA-approved rare disease drugs.
Agios is a Cambridge, Massachusetts biotech that reinvented itself around a single molecule. The company made its name in cancer metabolism, then sold its entire oncology business in 2021 to bet everything on rare genetic diseases of the red blood cell, with a pill called mitapivat at the center. Mitapivat activates pyruvate kinase, an enzyme red blood cells use to make energy; when the enzyme runs better, fragile red cells live longer and anemia improves.
That one mechanism has now produced two approved medicines. Pyrukynd (mitapivat) won FDA approval in 2022 for pyruvate kinase deficiency, and in December 2025 the same molecule was approved as Aqvesme for anemia in adults with alpha- or beta-thalassemia, reaching U.S. patients in early 2026. The company's biggest swing is sickle cell disease: in the Phase 3 RISE UP study, mitapivat met its hemoglobin-response endpoint but its reduction in pain crises did not reach statistical significance, so on May 12, 2026 Agios applied for accelerated approval based on the hemoglobin benefit. In July 2026 the FDA accepted the application with priority review for people 16 and older, and its decision is due November 1, 2026.
Agios decided not to advance tebapivat, a next-generation pyruvate kinase activator, after Phase 2 results in lower-risk myelodysplastic syndromes on May 29, 2026 and in sickle cell disease on July 21, 2026. Its earlier pipeline includes AG-181 for phenylketonuria, now in a Phase 1b trial in adults. For patients with rare anemias, Agios represents something unusual: a company betting that fixing red-cell energy metabolism can treat a whole family of diseases with one oral mechanism.
Agios Pharmaceuticals Drug Pipeline
Agios Pharmaceuticals has 13 active clinical trials across 4 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Agios Pharmaceuticals Clinical Trials (13)
Active and recruiting clinical trials sponsored by Agios Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Agios Pharmaceuticals FDA-Approved Drugs (2)
Medications developed or marketed by Agios Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
Agios Pharmaceuticals Trial Locations
Agios Pharmaceuticals clinical trials are running at 413 sites in 32 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (6)
Diseases targeted by Agios Pharmaceuticals's clinical trial and drug development programs
Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells. Immature blood cells do not develop properly and either die in the bone mar...
Phenylketonuria is an inherited metabolic disorder caused by a missing enzyme that prevents the body from properly breaking down the amino acid phenylalanine. This causes a buildup that can damage the...
Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk. Most pat...
Pyruvate kinase deficiency is an inherited red blood cell disorder in which a missing or weak enzyme leaves red cells short of energy, so they break down early and cause lifelong anemia, jaundice and ...
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes severe anemia, organ damage from iron overload, and bone abnormalities. Severe form...
Patient Resources
Organizations and resources related to Agios Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Agios Pharmaceuticals
Common questions about Agios Pharmaceuticals's rare disease programs, clinical trials, and treatments.