CAMBRIDGE, Mass., June 28, 2021 (GLOBE NEWSWIRE) – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in cellular metabolism developing and providing innovative treatments for genetically defined diseases, today announced it has submitted a Marketing Authorization Application (MAA) for mitapivat to the European Medicines Agency (EMA) for the treatment of adults with pyruvate kinase (PK) deficiency in the European Union. This submission follows the company’s recent New Drug Application (NDA) submission of mitapivat to the United States Food and Drug Administration (FDA) for the treatment of adults with PK deficiency in the United States.
“With our two cases, NDA and MAA, we are poised to provide the first potentially disease-modifying treatment for people with PK deficiency, lifelong chronic hemolytic anemia characterized by severe complications affecting multiple organs,” said said Chris Bowden, MD, chief medical officer. officer in Agios. “There is no approved treatment for PK deficiency, and current management strategies for blood transfusions and splenectomy are associated with short- and long-term risks, including iron overload, blood clots, and increased risk of infections. PK deficiency can lead to chronic fatigue, hemolytic crisis, gallstones, splenomegaly, cirrhosis of the liver, pulmonary hypertension and osteoporosis, and the burden of disease can adversely affect the ability of patients. patients navigate work and other daily activities, as well as their mental health. We look forward to working with the FDA and EMA to provide a potential new treatment option for this community that could help address the significant unmet needs of patients with PK deficiency.
Like the NDA, the submission of the MAA is based on the results of two pivotal studies, ACTIVATE and ACTIVATE-T, carried out respectively in non-transfused and regularly transfused adults with PK deficiency. A comprehensive analysis of this data – including Patient Reported Outcomes (PRO) – was recently presented at the European Hematology Association (EHA) Virtual Congress. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to assess the long-term safety, tolerability and efficacy of mitapivat treatment.
The use of Mitapivat is not approved by any regulatory authority.
About PK Deficiency
Pyruvate kinase (PK) deficiency is a rare inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells. Hereditary mutations in PKR genes cause an energy deficit in red blood cells, as evidenced by lower enzymatic activity of PK, lower levels of adenosine triphosphate (ATP) and an accumulation of upstream metabolites, including 2, 3-DPG (2,3-diphosphoglycerate).
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 load. . A PK deficiency can also cause quality of life issues, including issues 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 short and long term risks. There is currently no approved treatment for PK deficiency. For more information, please visit www.knowpkdeficiency.com.
Agios, in partnership with PerkinElmer Genomics, launched the Anemia ID program to offer free genetic testing to eligible patients in the United States with suspected hereditary anemia, including PK deficiency. The program was created in response to feedback from patients, advocates and physicians on the need for improved diagnostics to inform disease management decisions. To learn more, please visit www.AnemiaID.com.
Agios is focused on the discovery and development of new experimental drugs to treat genetically defined diseases through scientific leadership in the field of cell metabolism. The company’s most advanced drug candidate is a first-class pyruvate kinase R (PKR) activator, mitapivat, currently under evaluation for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has several new experimental therapies in clinical and preclinical development. For more information, please visit the company’s website at www.agios.com.
Caution regarding forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include those regarding the potential benefits of Agios’ products and product candidates; Agios’ plans for future regulatory submissions; and the potential benefits of Agios’ strategic plans and direction. The words âanticipateâ, â” ” ” ” expect, ‘intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to many important factors, risks and uncertainties which may cause actual events or results to differ materially from Agios’ current expectations and beliefs. Management’s expectations and, accordingly, all forward-looking statements contained in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation, risks and uncertainties related to: the impact of the COVID-19 pandemic on Agios’ planned activities, operations, strategy, objectives and milestones, including its ongoing and planned research activities, its ability to conduct clinical trials in current and planned, clinical supply of current or future drug candidates, commercial supply of future approved products and the launch, market and sale of future approved products; the results of clinical trials and preclinical studies of Agios, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the US FDA, EMA or other regulatory authorities, clinical trial site investigation review boards, and publication review bodies; Agios’ ability to obtain and maintain required regulatory approvals and enroll patients in its planned clinical trials; unforeseen cash flow needs and expenses and competitive factors; Agios’ ability to obtain, maintain and enforce patents and other intellectual property protections for all product candidates it develops; Agios’ ability to establish and maintain collaborations; and general economic and market conditions. These and other risks are further described under the heading “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission, or SEC, including the risks and uncertainties set forth under the heading “Risk Factors”. risk in our documents filed with the SEC. . Although the list of factors presented here is considered representative, this list should not be taken as a complete statement of all potential risks and uncertainties. All forward-looking statements contained in this communication are made only as of the date hereof, and we assume no obligation to update any forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation. to do so other than what may be required by law.