- Trial Phase
- Approved (2 indications) / Filed for SCD (Phase 3 completed)
- Time to Market
- Already approved for PK deficiency (2022) and thalassemia (Dec 2025). Sickle cell disease sNDA under FDA Priority Review with PDUFA November 1, 2026.
- Why 8/10
- The next hard, dated catalyst is the FDA PDUFA goal date of November 1, 2026 for accelerated approval of mitapivat in sickle cell disease (sNDA granted Priority Review on July 7, 2026). This is a ~4-month binary catalyst on the company's lead asset, which, if approved, would open a large new market. ImpactScore 8 reflects this timebound binary on the primary value driver.
Mitapivat is already generating revenue ($20.7M in Q1 2026) from PK deficiency and thalassemia approvals, but the stock's next big swing is the November 1, 2026 PDUFA for sickle cell disease. The RISE UP data was mixed (hemoglobin win, pain-crisis miss), so approval is uncertain. Watch for FDA AdCom scheduling and any pre-PDUFA news flow. The stock has an RSI-14 of 73.6 (overbought) after running up on the Priority Review grant — some consolidation before the binary could present a better entry.
Price Chart
Executive Summary
Mitapivat is an oral pyruvate kinase (PK) activator developed by Agios Pharmaceuticals (AGIO) — already approved and commercialized in two rare blood-disease indications (PK deficiency as PYRUKYND, 2022; thalassemia as AQVESME, Dec 2025). Q1 2026 revenue more than doubled to $20.7M with 242 thalassemia prescriptions written. The defining near-term catalyst is the November 1, 2026 PDUFA date for accelerated approval in sickle cell disease — a vastly larger market opportunity. The RISE UP Phase 3 data were mixed: strong hemoglobin and transfusion-burden results, but the pain-crisis primary endpoint missed. Approval would unlock peak sales estimated at $1B+. The stock trades at ~$37-42 with 12 buy/strongBuy ratings, 4 holds, 0 sells, and a mean analyst target around $40.75. A boxed warning for liver injury and a REMS program apply to the thalassemia label.
How It Works
Mitapivat is a pill taken by mouth that activates a key enzyme (pyruvate kinase) in red blood cells. This enzyme helps red blood cells produce energy and survive longer. In certain genetic blood disorders (PK deficiency, thalassemia, sickle cell disease), red blood cells break down too quickly. By boosting the enzyme's activity, mitapivat helps red blood cells live longer, reducing anemia and the need for blood transfusions.
Trial Status
Mitapivat is FDA-approved for hemolytic anemia in adults with pyruvate kinase (PK) deficiency (February 2022, brand PYRUKYND) and for anemia in adults with alpha- or beta-thalassemia (December 2025, brand AQVESME). The RISE UP Phase 3 trial in sickle cell disease (SCD) toplined in November 2025 with mixed results: statistically significant hemoglobin response (40.6% vs 2.9% placebo) and reduced transfusion burden, but the primary endpoint of sickle cell pain crisis reduction missed statistical significance. Based on these data, an sNDA for accelerated approval in SCD was submitted in May 2026, granted FDA Priority Review in July 2026 with a PDUFA of November 1, 2026.
The Two-Sided Case
Upcoming Catalysts
- PDUFA goal date for mitapivat sNDA in sickle cell disease: November 1, 2026 (FDA Priority Review, accelerated approval pathway).
- Potential FDA Advisory Committee meeting for mitapivat in SCD (ahead of PDUFA — no date announced yet).
- Q2 2026 earnings (late July/early August 2026) — will show AQVESME prescription growth trajectory.
- Phase 2b tebapivat trial in LR-MDS topline results expected H1 2026 (pipeline asset, not mitapivat).
Source Filing
The SecBot filing or press-release report that triggered this drug report:
Agios Pharmaceuticals, Inc. Press Release AGIO ·Originating report