About Us
Over 7,000 rare diseases affect millions worldwide, and many have limited or no approved treatments. At Agios, we are striving to change that.
For over two decades, Agios has built a strong and enduring foundation rooted in scientific innovation. We are fueled by the power of connection – to one another, to our communities, and to the rare disease patients we serve. For us, progress begins with truly listening to patients, caregivers, and healthcare providers. This understanding enables us to deliver meaningful innovation and develop therapies with the potential to change lives.
In 2022, our first rare disease therapy, PYRUKYND® (mitapivat), was approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the U.S., and for PK deficiency in adults in the European Union and Great Britain. Now, Agios is actively pursuing regulatory approval for mitapivat for the treatment of adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates. Mitapivat is also being investigated in global Phase 3 trials for sickle cell disease and pediatric PK deficiency. Beyond mitapivat, we have a robust early- and mid-stage pipeline exploring treatments for myelodysplastic syndromes, sickle cell disease, phenylketonuria and polycythemia vera.
To learn more, visit us at www.agios.com.
