Agios Pharmaceuticals, Inc. announced that the Saudi Food and Drug Authority (SFDA) has approved PYRUKYND (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
“The SFDA’s decision marks a significant milestone, as Saudi Arabia becomes the first country to approve PYRUKYND for the treatment of adult patients with thalassemia, offering new hope to a community that has long faced debilitating, often life-threatening symptoms with limited or no therapeutic options,” said Brian Goff, Chief Executive Officer, Agios. “Given the substantial burden and high estimated prevalence of thalassemia in Saudi Arabia, we are proud to partner with NewBridge Pharmaceuticals – a company specializing in delivering innovative treatments across the Middle East and North Africa – to help ensure PYRUKYND is accessible to these patients. We look forward to bringing PYRUKYND to more thalassemia patients globally and are actively preparing for potential launches in the U.S., United Arab Emirates, and Europe.”
The SFDA approval of PYRUKYND in thalassemia is based on the results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. The New Drug Application (NDA) for PYRUKYND was accepted under the SFDA’s Breakthrough Medicines Program, a program that aims to facilitate and accelerate the development and review of new medicines that address unmet medical need in the treatment of serious or life-threatening conditions.
In 2024, Agios entered into a distribution agreement with NewBridge Pharmaceuticals to advance regulatory filings and commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region, which includes Saudi Arabia, United Arab Emirates, Kuwait, Qatar, Oman, and Bahrain. NewBridge Pharmaceuticals was founded in 2010 to be a first-in-class commercialization platform for innovative therapeutics developed by global pharmaceutical companies, with a mission to bridge the access gap in the Middle East and North Africa.
“Thalassemia is a rare, lifelong genetic disorder that causes chronic anemia and can result in severe complications, including organ damage and cardiac disease,” said Ali Taher, M.D., Ph.D., Professor of Medicine, Hematology & Oncology, and Director of Naef K. Basile Cancer Institute, American University of Beirut Medical Center in Beirut, Lebanon. “Until now, treatment options have been limited and often come with serious risks. The findings from the ENERGIZE and ENERGIZE-T trials support PYRUKYND as a disease-modifying, oral therapy for thalassemia – offering a much-needed new option to address the critical needs of this patient population.”
“Historically, treatment options have been limited and are often specific to certain thalassemia subtypes or transfusion needs, leaving too many patients underserved,” said Khaled Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates. “With this approval, PYRUKYND is now indicated in Saudi Arabia for all adult patients with alpha- or beta-thalassemia, regardless of transfusion dependency status. This broad indication represents a truly meaningful advancement for the entire thalassemia community.”
Regulatory applications for PYRUKYND in adult patients with thalassemia are under review by health authorities in the U.S., United Arab Emirates, and European Union. The Prescription Drug User Fee Act (PDUFA) goal date assigned by the U.S. Food and Drug Administration is September 7, 2025.
PYRUKYND is also approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the U.S., and for the treatment of PK deficiency in adult patients in the European Union and Great Britain.