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Just saw that Agios got PYRUKYND approved in the UAE for thalassemia patients. Interesting move - it's the only drug approved there for this specific thalassemia profile, which is kind of a big deal for patients who actually need it.
The approval is based on their Phase 3 trials (ENERGIZE and ENERGIZE-T), so at least it's backed by solid data. They already have it approved in the US and Europe, and apparently they partnered with NewBridge Pharmaceuticals back in 2024 to push it through the GCC region - Saudi Arabia, UAE, Kuwait, Qatar, Oman, Bahrain.
What's interesting is they're still waiting on European Commission review for the thalassemia indication there. Meanwhile in the US it's already out as AQVESME. So basically they're expanding geographically, which makes sense if the thalassemia profile shows real benefits. Not sure how many patients this actually impacts though.