1차 출처 기록
FDA가 겸상적혈구병에 대한 mitapivat sNDA에 대해 우선심사(Priority Review)를 부여하고 2026년 11월 1일을 PDUFA 목표일로 설정했습니다. 이 신청은 가속승인 경로(accelerated approval pathway)로 제출되었으며, 임상적 유효성을 확인하기 위해 확증적 3상 시험(REIGNITE)이 필요합니다. 이는 잠재적인 새로운 적응증에 대한 단기 규제 결정 시점을 확립합니다.
EX-99.1 2 agio-73026xfyxex991earning.htm EX-99.1 Document Agios Reports Second Quarter 2026 Financial Results and Provides Business Update • Mitapivat (PYRUKYND® and AQVESME™) delivered worldwide net revenues of $44.7 million in the second quarter of 2026, compared to $12.5 million in the second quarter of 2025 • Strong U.S. commercial launch of AQVESME in thalassemia, with 442 cumulative prescriptions written as of June 30, 2026 • sNDA for mitapivat granted FDA Priority Review in sickle cell disease with PDUFA goal date of November 1, 2026 • Diversified and expanded late-stage pipeline with licensing of cevidoplenib for immune thrombocytopenia and advancement of AG-236 into Phase 2/3 development for polycythemia vera • $964.8 million in cash, cash equivalents, and marketable securi