Agios Pharmaceuticals Touts Strong AQVESME Launch as Sickle Cell Filing Advances

Key Points

• Agios said its rare hematology strategy is gaining momentum, pointing to a “very strong” early launch for AQVESME in thalassemia and a recently filed supplemental NDA for mitapivat in sickle cell disease.
• The company said the sickle cell filing is advancing under an accelerated approval approach, with FDA alignment on a confirmatory trial focused on transfusion independence in 159 patients over one year.
• Commercially, Agios reported 242 first-quarter prescriptions for AQVESME from REMS-certified physicians and said the launch is broad-based, while keeping its $1 billion peak sales guidance intact for thalassemia and pyruvate kinase deficiency.

Agios Pharmaceuticals NASDAQ: AGIO executives said the company is focused on advancing mitapivat across rare hematologic diseases, highlighting early launch traction in thalassemia and a recently submitted supplemental new drug application for sickle cell disease during a presentation at the 2026 RBC Capital Markets Global Healthcare Conference.

Brian Goff, Agios’ chief executive officer, said the company has had “a really great start to 2026,” citing the launch of AQVESME, the brand name for mitapivat in thalassemia, and the filing of an sNDA with the U.S. Food and Drug Administration for mitapivat in sickle cell disease.

Goff said Agios is continuing to sharpen its focus on rare hematology and is seeking “durable value creation for the long term.” He described the first quarter of AQVESME’s launch as “very strong,” while noting that it remains early in the launch cycle.

Sickle Cell Filing Moves Forward Under Accelerated Approval Pathway

Sarah Gheuens, Agios’ chief medical officer, said the company held a pre-sNDA meeting with the FDA to discuss data from RISE UP, the company’s sickle cell disease trial. The trial had two primary endpoints, and Gheuens said the hemoglobin endpoint enabled discussion with the agency about an accelerated approval pathway.

Gheuens said the data package included “a lot of other clinical meaningful data,” including evidence supporting mitapivat’s anti-hemolytic profile and additional benefit among hemoglobin responders. She said Agios has reached agreement with the FDA on a confirmatory trial required for accelerated approval.

The confirmatory trial will be focused on transfusion independence, will include 159 patients and will run for one year, Gheuens said. She described it as “streamlined” and “simple” for patients to participate in. The trial will also lower the age range to 12 years and older, which Gheuens said is intended to support pediatric development.

Gheuens said more details on the confirmatory trial and the RISE UP data supporting the sNDA will be presented at the European Hematology Association meeting in Stockholm next month.

Asked whether Agios expects priority review or standard review, Gheuens said that decision rests with the FDA. However, she emphasized the high unmet need in sickle cell disease and said “there is an urgency” for new treatment options.

Company Says Novo Data Does Not Change Its Pathway

Gheuens said recent top-line data from Novo Nordisk does not affect Agios’ regulatory package or the accelerated approval pathway it is pursuing. She said Agios remains confident in its own data set, noting consistent evidence of hemoglobin response, anti-hemolytic effect and additional clinical benefit that will be further discussed at EHA.

Goff added that Agios’ application is an sNDA and said the company has had multiple interactions with the FDA over several years across hemolytic anemias, including mitapivat approvals in pyruvate kinase deficiency and thalassemia.

On the potential label for sickle cell disease, Gheuens said Agios has “optionality.” AQVESME, approved for thalassemia, has a REMS program and uses a 100-milligram dose, while PYRUKYND is approved for pyruvate kinase deficiency without a REMS. Gheuens said mitapivat’s safety profile in sickle cell disease “may not warrant a REMS,” but said that will be subject to negotiation with regulators.

Commercial Team Highlights Thalassemia Launch

Tsveta Milanova, Agios’ chief commercial officer, said the company is encouraged by the early reception of AQVESME in thalassemia. Agios reported 242 prescriptions in the first quarter from REMS-certified physicians.

Milanova said prescriptions came from clinicians across the country and were not concentrated among a small number of prescribers. She said the launch showed breadth across patient types, including transfusion-dependent, non-transfusion-dependent, alpha thalassemia and beta thalassemia patients. However, she said a large proportion of prescriptions came from transfusion-dependent patients, which she attributed to their more frequent interaction with the healthcare system.

Over time, Milanova said Agios expects to penetrate more deeply into the non-transfusion-dependent thalassemia segment, which she described as two-thirds of the opportunity. She said demand growth may moderate as the company moves beyond early, highly engaged patients and prescribers.

Milanova said Agios remains comfortable with its previously stated $1 billion global peak sales guidance for pyruvate kinase deficiency and thalassemia, with the majority expected to be driven by thalassemia in the U.S.

Agios executives indicated the company is likely to continue providing prescription updates in the near term, while noting that revenue will eventually become the more relevant metric as the launch matures.

Pricing and Access Considerations for Sickle Cell Disease

Milanova said Agios will provide more information on sickle cell disease pricing closer to a potential approval, when the company can account for the final label and competitive environment. She said sickle cell disease has a higher proportion of Medicaid coverage than thalassemia or pyruvate kinase deficiency, which could affect net pricing because of mandatory Medicaid rebates.

She estimated that Medicaid coverage in sickle cell disease can be about 50% of the patient population, though she said data varies and is age dependent. By contrast, she said most thalassemia and pyruvate kinase deficiency patients are commercially insured.

Milanova also discussed the time from prescription to treatment initiation for AQVESME, saying Agios initially guided to 10 to 12 weeks. In the first quarter, the timeline was shorter because early prescribers and patients were highly engaged. Over time, she said the company expects the timeline to move closer to 10 to 12 weeks, driven by prior authorization processes and liver testing requirements under the REMS.

Pipeline Update Includes MDS Program

Gheuens also addressed tebapivat in myelodysplastic syndromes, describing the opportunity as “high risk, high reward.” She said the risk stems from MDS being the first non-hemolytic anemia in which Agios is studying PK activation, while the potential reward reflects the size of the market and the need for additional options for patients who outlive current therapies.

Asked what part of the Agios story is most underappreciated, Goff pointed to sickle cell disease. He cited the high unmet need, the strength of the RISE UP data, the filed sNDA and Agios’ regulatory and commercial preparation as factors he believes investors may not fully recognize.

About Agios Pharmaceuticals NASDAQ: AGIO

Agios Pharmaceuticals, Inc is a biopharmaceutical company founded in 2008 as a spin-out from research at Dana-Farber Cancer Institute and the Broad Institute. Headquartered in Cambridge, Massachusetts, Agios focuses on understanding and targeting cellular metabolism to develop novel therapies for cancer and rare genetic diseases. The company's scientific platform integrates genomic discovery, metabolic profiling and precision medicine approaches to identify and advance small-molecule candidates that correct or exploit metabolic dysfunction.

Agios's lead products are IDH (isocitrate dehydrogenase) inhibitors that target specific cancer mutations.

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