First and Only Disease-modifying Therapy for EU Patients with Rare Blood Disorder
CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, today announced that the European Commission (EC) has granted marketing authorization for PYRUKYND® for the treatment of PK deficiency in adult patients. PYRUKYND® is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the EU with this rare, debilitating, lifelong hemolytic anemia.
“People with PK deficiency suffer from a lifetime of chronic anemia, associated complications and symptoms that can be detrimental to their work, family and social lives,” said Andreas Glenthøj, hematologist and head of the Danish Center for Hemoglobinopathies at Rigshospitalet, and associate professor at the University of Copenhagen. “PYRUKYND® offers new hope for this community, and I am honored to have contributed to the research efforts that enabled the approval of the first therapy for adults with PK deficiency in the EU.”
“With today’s EU approval, we are proud to expand the positive impact of PYRUKYND® for more patients with PK deficiency around the globe,” said Brian Goff, chief executive officer at Agios. “We are dedicated to continued innovation on behalf of people with rare and genetically defined diseases, and are working to further expand the impact of PYRUKYND® through our ongoing investigational pivotal programs in pediatric PK deficiency, thalassemia and sickle cell disease.”
Agios is providing access to PYRUKYND® for the treatment of PK deficiency in adults receiving care in the EU through a global managed access program. More details about this program can be found on Agios.com.
PYRUKYND® was previously granted orphan drug designation by the EMA, which is maintained at the time of EU marketing authorization. Agios has also applied for a marketing authorization for PYRUKYND® as a treatment for PK deficiency in adult patients in Great Britain under the European Commission Decision Reliance Procedure (ECDRP) with the Medicines and Healthcare Products Regulatory Agency (MHRA).
PYRUKYND® was approved by the U.S. Food and Drug Administration (FDA) in February 2022 for the treatment of hemolytic anemia in adults with PK deficiency.
PYRUKYND® Safety and Efficacy DataThe EU marketing authorization was based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.
- The Phase 3 ACTIVATE trial of mitapivat achieved its primary endpoint. PYRUKYND® demonstrated a statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused.
- 40 percent (n=16) of patients randomized to PYRUKYND® achieved a hemoglobin response, compared to 0 patients randomized to placebo (2-sided p