Agios Extends Mitapivat Sickle-Cell PDUFA to February 2027

The Tip Desk · November 7, 2026 · 3 min read

Worldwide mitapivat net revenue reached $55.4 million in the third quarter, more than four times a year earlier, as U.S. AQVESME demand kept building.

Agios Pharmaceuticals (AGIO) said the Food and Drug Administration 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, after classifying the company’s response to a REMS information request as a major amendment.

The commercial-stage biopharmaceutical company said it remains launch-ready and is in labeling discussions with the FDA, including elements of a potential REMS. Chief Executive Officer Brian Goff said the third quarter was a record for product revenue, outperforming the entire 2025 total in three months, and that the U.S. commercial launch of AQVESME in thalassemia had built the commercial foundation for sickle cell disease.

Worldwide net revenue for mitapivat, sold as PYRUKYND and AQVESME, was $55.4 million in the third quarter of 2026, more than four times the $12.9 million reported in the third quarter of 2025. U.S. net revenue was $48.7 million, up 20% sequentially from $40.9 million in the second quarter, which the company attributed to the late-January U.S. launch of AQVESME in thalassemia. Ex-U.S. net revenue was $6.6 million, up 74% sequentially from $3.8 million, driven primarily by early access programs in Europe after PYRUKYND’s late-May 2026 European Union approval for thalassemia. The company is advancing those markets through partnerships with Avanzanite Bioscience B.V. in Europe and NewBridge Pharmaceuticals in the Gulf Cooperation Council region.

In the third quarter, 177 AQVESME prescriptions with completed Agios start forms were written by REMS-certified U.S. physicians, bringing cumulative prescriptions to 619 as of September 30, 2026, versus 442 as of June 30. An additional 48 cumulative prescriptions had start forms in process and were not included in that metric. Agios said demand continued to broaden, with deeper penetration in transfusion-dependent patients and expansion into the larger non-transfusion-dependent population, and that approximately 75% of patients remained on AQVESME after six months of treatment.

Net loss was $65.0 million, or $1.09 a share, compared with net loss of $103.4 million, or $1.78 a share, a year earlier. Loss from operations was $77.2 million. Cost of sales was $2.5 million. Research and development expenses were $73.5 million, down from $86.8 million a year earlier, which the company said was driven primarily by reductions in spend associated with mitapivat and tebapivat process development and certain clinical trials. Selling, general and administrative expenses were $56.5 million, up from $41.3 million a year earlier, due to an increase in activities supporting the U.S. commercial launch of AQVESME in thalassemia and preparation for the potential U.S. commercial launch of mitapivat in sickle cell disease.

Cash, cash equivalents and marketable securities were $907.0 million as of September 30, 2026, down from $964.8 million as of June 30, 2026. Agios said that cash, together with anticipated product revenue and interest income, will provide the financial independence to execute the AQVESME launch, prepare for the potential sickle cell launch, and progress the pipeline. The company’s goal is to achieve non-GAAP profitability in 2028.

Over the course of 2026, Agios reviewed its operating model and said it will have reduced R&D headcount by over 100 roles by year-end, a more than 35% decrease compared with 2025. The company expects the associated cost savings to be realized in 2027.

Fourteen abstracts led by Agios and external collaborators were accepted for ASH 2026, including three oral presentations with new data across mitapivat clinical programs. New RISE UP Phase 3 data in sickle cell disease showed durable hemoglobin improvements for up to 115 weeks with continued treatment. First clinical data for AG-236 from a Phase 1 trial in healthy volunteers supported every-six-month dosing in polycythemia vera.