Agios Secures Saudi Food and Drug Authority (SFDA) Approval For PYRUKYND In Thalassemia, Strengthening GCC Access

Agios Pharmaceuticals, Inc. announced that the Saudi Food and Drug Authority (SFDA) has approved PYRUKYND® (mitapivat) for the treatment of adults with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. This marks a major milestone in expanding access to treatments for rare blood disorders in the Middle East.

“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. 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,” said Brian Goff, Chief Executive Officer, Agios. 

The approval is based on data from the pivotal global Phase 3 trials, ENERGIZE and ENERGIZE-T, which evaluated mitapivat’s efficacy and safety in adult thalassemia patients. The drug application was accepted under the SFDA’s Breakthrough Medicines Program, an initiative aimed at expediting the review of therapies addressing high unmet medical needs.

“Thalassemia is a rare, lifelong genetic disorder that causes chronic anemia and can result in severe complications, including organ damage and cardiac disease. 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,” 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.

To support regional access, Agios partnered with NewBridge Pharmaceuticals in 2024 to manage regulatory filings and commercialization efforts across the GCC, including Saudi Arabia and neighboring countries. PYRUKYND is also under regulatory review in the U.S., EU, and UAE, with a PDUFA decision from the FDA expected by September 7, 2025. The drug is already approved for treating pyruvate kinase deficiency in adults in the U.S., EU, and U.K.

“Historically, treatment options have been limited and are often specific to certain thalassemia subtypes or transfusion needs, leaving too many patients underserved. 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,” said Khaled Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates.