The Saudi Food and Drug Authority (SFDA) has approved Agios Pharmaceuticals’ Pyrukynd (mitapivat) for adults with alpha or beta-thalassemia.
Pyrukynd is specifically indicated for haemolytic anaemia in adults with pyruvate kinase deficiency.
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The approval was based on the ENERGIZE and ENERGIZE-T Phase III trial data in both non-transfusion-dependent and transfusion-dependent forms of thalassemia.
Pyrukynd’s new drug application benefited from the SFDA’s breakthrough medicines programme, reflecting its potential to address critical unmet medical needs.
The therapy is available in oral tablet form in 5mg, 20mg and 50mg strengths.
Agios CEO Brian Goff stated: “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.
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By GlobalData“Given the substantial burden and high estimated prevalence of thalassemia in Saudi Arabia, we are proud to partner with NewBridge Pharmaceuticals – a company specialising 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 US, UAE, and Europe.”
Agios and NewBridge Pharmaceuticals signed a distribution agreement in 2024. This partnership focused on regulatory filings and commercialisation efforts within the Gulf Cooperation Council region, including in the United Arab Emirates (UAE), Saudi Arabia, Qatar, Kuwait, Oman and Bahrain.
Health authorities across jurisdictions are currently reviewing regulatory applications for Pyrukynd as a therapeutic option for adults with thalassemia. These include agencies in the US, the UAE and the European Union.
The US Food and Drug Administration has allotted a Prescription Drug User Fee Act goal date of September 2025.
