Mitapivat提升了镰状细胞患者的血红蛋白, 但并未减轻疼痛或疲劳, 但Agios却在安全和血红蛋白增益的基础上寻求美国批准。 Mitapivat boosted hemoglobin in sickle cell patients but didn’t reduce pain or fatigue, yet Agios seeks U.S. approval based on safety and hemoglobin gains.

镰状细胞病中mitapivat的一项三期试验显示，该药在40.6%的患者中显著增加血红蛋白，而安慰剂组为2.9%，但未显著减少疼痛危机或患者报告的疲劳。 A Phase 3 trial of mitapivat in sickle cell disease showed the drug significantly increased hemoglobin in 40.6% of patients versus 2.9% on placebo, but did not significantly reduce pain crises or patient-reported fatigue. 尽管结果好坏参半,但Agios计划以安全和血红蛋白改善为由,寻求美国批准,预计在2026年林业发展局会议之后提交监管文件。 Despite mixed results, Agios plans to seek U.S. approval, citing safety and hemoglobin improvements, with a regulatory submission expected after a 2026 FDA meeting.