Agios Pharmaceuticals has announced that the European Commission (EC) has granted orphan medicinal product designation to their oral therapy, mitapivat, for treating sickle cell disease (SCD). Already recognized with orphan drug status by the US FDA in November 2020, mitapivat is being investigated for its potential benefits in addressing this serious condition. Currently marketed as PYRUKYND in the US and EU for hemolytic anemia, mitapivat’s safety and efficacy for SCD are under evaluation through the ongoing Phase II and III RISE UP trials. Sarah Gheuens, head of R&D at Agios, emphasized the importance of this development and highlighted the need for innovative treatments for sickle cell disease. Results from the Phase III study are expected to be shared with the community by late 2025.
At OxyDial, we are encouraged by Agios’s progress in fighting sickle cell disease. We remain hopeful for the transformative potential of ongoing research efforts in addressing the complexities of SCD.
Read more: https://lnkd.in/eCxpaJAF
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