Agios Receives Positive CHMP Opinion for PYRUKYND® (mitapivat) for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients
CAMBRIDGE, Mass., Sept. 16, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion on September 15, 2022, recommending the granting of a marketing authorization for PYRUKYND® (mitapivat) for the treatment of pyruvate kinase (PK) deficiency in adult patients. PK deficiency is a rare, debilitating, lifelong hemolytic anemia. PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy for European patients with PK deficiency.
“The positive CHMP opinion for PYRUKYND® is a landmark event for European patients with PK deficiency, who currently have no disease-modifying treatment options,” said Eduard J. van Beers, M.D., Ph.D., hematologist and associate professor at University Medical Center Utrecht. “PK deficiency is characterized by severe symptoms and complications, regardless of transfusion status, and has a significant impact on patients’ quality of life. If approved, I look forward to having an oral medication available that has demonstrated clinically meaningful impact for patients with a range of PK deficiency symptoms and presentations.”
“Today’s positive CHMP opinion is an important step toward our goal of expanding PYRUKYND® access for adults with PK deficiency around the world,” said Sarah Gheuens, M.D., Ph.D., head of R&D and chief medical officer at Agios Pharmaceuticals. “In addition to this milestone, we remain focused on our global clinical development programs for PYRUKYND® in thalassemia, sickle cell disease and pediatric PK deficiency and look forward to expanding the impact of this medication to even more patients.”
PYRUKYND® was previously granted orphan drug designation by the EMA.
PYRUKYND® Safety and Efficacy Data
A full analysis of these data was presented at the 2021 European Hematology Association (EHA) Virtual Congress, and results from ACTIVATE were published in the New England Journal of Medicine. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat; initial results from the extension study were presented at the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition.
The company is enrolling pediatric PK deficiency patients in two pivotal studies – ACTIVATE-kids and ACTIVATE-kidsT – in patients who are not regularly transfused and who are regularly transfused, respectively. Agios also continues to advance its Phase 3 ENERGIZE and ENERGIZE-T studies in not regularly transfused and regularly transfused adults with thalassemia, as well as its Phase 2/3 RISE UP study in sickle cell disease.
About PK Deficiency
PK deficiency is associated with serious complications, including gallstones, pulmonary hypertension, extramedullary hematopoiesis, osteoporosis and iron overload and its sequelae, which can occur regardless of the degree of anemia or transfusion burden. PK deficiency can also cause quality of life problems, including challenges with work and school activities, social life and emotional health. Current management strategies for PK deficiency, including red blood cell transfusions and splenectomy, are associated with both short- and long-term risks. For more information, please visit www.knowpkdeficiency.com.
About PYRUKYND® (mitapivat)
IMPORTANT SAFETY INFORMATION
Adverse Reactions: Serious adverse reactions occurred in 10% of patients receiving PYRUKYND in the ACTIVATE trial, including atrial fibrillation, gastroenteritis, rib fracture, and musculoskeletal pain, each of which occurred in 1 patient. In the ACTIVATE trial, the most common adverse reactions including laboratory abnormalities (=10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia.
Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.
Cautionary Note Regarding Forward-Looking Statements
Session Details TBA
Employee Engagement, Knowledge Management, and AI for the New Workplace
The Cyber Threat Landscape and How MSPs Can Stay in Control