27/12/2025
📢 BIG NEWS: A Historic Milestone for Thalassaemia Warriors! 🩸🌟
We have some AMAZING news from the global medical world! 🌍🔬 The U.S. FDA has just approved a brand-new medicine called AQVESME™ (mitapivat). 💊🎉
This is a HUGE win for our community because it is the first-ever pill 💊 approved to help adults with:
✅ Alpha (α) Thalassaemia 🧬
✅ Beta (β) Thalassaemia 🧬
✅ Transfusion-Dependent (TDT) 🩸💉
✅ Non-Transfusion-Dependent (NTDT) 💪
How does it work? 🤔💡
AQVESME is not an injection—it’s a daily pill! 💊 It works like a "rechargeable battery" 🔋 for your red blood cells. It helps them:
* 🛡️ Stay Stronger and live longer in your body.
* 📈 Boost Hemoglobin levels naturally.
* ⚡ Fight Fatigue so you have more energy for life! 🏃♂️💨
* 🩸 Reduce Transfusions – meaning fewer trips to the hospital! 🏥🏥
🛡️ Safety & Care 👩⚕️🩺
Because your health is the #1 priority, this medicine comes with a safety plan:
* 🧪 Liver Checks: Patients will have regular blood tests to make sure their liver stays healthy. ✅
* 📋 REMS Program: A special system to ensure doctors and patients use the medicine correctly. ✍️
🕒 When can we get it? 🗓️✈️
* 🇺🇸 USA: Expected in late January 2026.
* 🇪🇺 Europe: Approval is currently being processed. ⏳
* 🇱🇰 Sri Lanka: While it’s not here yet, this global approval is the first step to bringing this medicine to patients all over the world! 🌏🤝
💡 Quick FAQ Summary 📝
* How is it taken? 💊 2 times a day (100mg tablets).
* Does it remove iron? 🧲 Not directly, but by reducing transfusions, it helps stop new iron build-up! 🚫🧱
* Who can take it? 👨🦰👩🦱 Adults (18+) with Alpha or Beta Thalassaemia.
🌟 We are moving toward a brighter, stronger future together! 🌟
🇱🇰 💪 🔬 🌈 🩸 💊 🧬
🎉 Major Milestone for the Global Thalassaemia Community
The U.S. Food and Drug Administration (FDA) has approved ™ ( ), developed by Agios Pharmaceuticals, for the treatment of anaemia in adult with α- or β- — covering both transfusion-dependent (TDT) and non-transfusion-dependent (NTDT) forms of the disorder.
This marks the first-ever therapy indicated across both patient groups, representing a truly historic advance in the management of this complex hereditary .
AQVESME is an oral pyruvate kinase (PK) activator, shown in the global Phase III ENERGIZE and ENERGIZE-T clinical studies to deliver statistically significant and clinically meaningful improvements in and , as well as reductions in compared to placebo among 452 enrolled patients.
Brian Goff, Chief Executive Officer of Agios, stated: “Today is a landmark moment for the thalassemia community, bringing forward an innovative, disease-modifying oral medicine to address the urgent needs of people living with this devastating rare blood disorder.”
📅 In the US, the therapy is expected to be launched in late January 2026 following implementation of the Risk Evaluation and Mitigation Strategy (REMS) safety monitoring programme. Meanwhile, approval in the is pending, and additional research is exploring mitapivat’s potential in as well.
To learn more, see the full official announcement in the first comment.
