Agios pushes back mitapivat sickle cell decision to February 2027

Agios Pharmaceuticals said on November 5, 2026 that the FDA has extended the PDUFA goal date for its mitapivat sickle cell disease supplemental New Drug Application by three months, from November 1, 2026, to February 1, 2027.¹ The delay followed a regulatory back-and-forth: the FDA had requested additional information to evaluate whether a REMS is necessary for mitapivat in sickle cell disease, and classified Agios's response to that request as a major amendment to the sNDA, triggering the three-month extension.¹ Agios said it will keep discussing labeling with the FDA during the ongoing review, including possible REMS provisions.¹

On the commercial side, mitapivat (sold as PYRUKYND and AQVESME) brought in worldwide net revenues of $55.4 million in the third quarter of 2026, more than four times the $12.9 million reported in the third quarter of 2025.¹ U.S. uptake of AQVESME in thalassemia continued to build, with 177 prescriptions with completed Agios start forms written by REMS-certified U.S. physicians in the quarter, bringing cumulative prescriptions to 619 as of September 30, 2026,¹ plus an additional 48 cumulative prescriptions with start forms in process as of the same date.¹

Agios also outlined data expected at the American Society of Hematology meeting. The company said 14 abstracts led by Agios and external collaborators have been accepted for presentation at ASH 2026, taking place December 12-15, 2026 in New Orleans, including three oral presentations with new data across mitapivat programs.¹ Separately, new data from the RISE UP Phase 3 trial of mitapivat in sickle cell disease showed durable hemoglobin improvements for up to 115 weeks with continued treatment.¹

On operations, Agios disclosed it reviewed its operating model during 2026 and expects to cut its R&D headcount by more than 100 roles by year end, a reduction of over 35% versus 2025, with associated cost savings expected in 2027.¹ The company reported cash, cash equivalents and marketable securities of $907.0 million as of September 30, 2026, down from $1.2 billion at the end of 2025,¹ and said this funding, along with anticipated product revenue, is expected to support its launches and pipeline, with a goal of reaching non-GAAP profitability in 2028.¹