3 Hematology Headlines You Missed in July 2026

July included regulatory movement in sickle cell disease and a discontinued development program in the same condition.

The US Food and Drug Administration (FDA) granted priority review to Agios' mitapivat sNDA for sickle cell disease, with a PDUFA date of November 1, 2026, which, if approved, would become the first oral pyruvate kinase activator for the condition. About 2 weeks later, Agios discontinued development of tebapivat, a separate pyruvate kinase activator, in sickle cell disease after phase 2 results did not show sufficient differentiation from placebo. HCPLive also spoke with Wendy Stock, MD, about the FDA's approval of Orca-T (Tregzi), a precision-engineered cell therapy for allogeneic transplant in adults with hematological malignancies.

Below is a summary of the July 2026 stories in hematology, covering regulatory decisions and trial data in sickle cell disease and cell therapy for transplant-related graft-versus-host disease.

Regulatory News

FDA Grants Priority Review to Agios' Mitapivat sNDA for Sickle Cell Disease

On July 7, 2026, the FDA granted priority review to Agios' supplemental NDA for mitapivat, an oral pyruvate kinase activator, in sickle cell disease, with a PDUFA date set for November 1, 2026. Mitapivat is already approved in the US for two other hemolytic anemias, pyruvate kinase deficiency and thalassemia.

Agios Ends Tebapivat Development in Sickle Cell Disease After Phase 2

On July 21, 2026, Agios discontinued development of tebapivat in sickle cell disease after phase 2 results showed hemoglobin responses across active doses that were not sufficiently differentiated from placebo. The company said the decision was based on lack of differentiation rather than safety or tolerability concerns, and that it will continue to focus its sickle cell disease efforts on mitapivat.

Expert Perspective

FDA Approves Orca-T After Phase 3 Trial Improves Chronic GVHD-Free Survival

In this interview, Wendy Stock, MD, discusses the FDA approval of Orca-T (Tregzi), a precision-engineered allogeneic regulatory T-cell therapy for adults with hematological malignancies undergoing matched-donor transplant. The conversation covers phase 3 data showing improved chronic graft-versus-host disease-free survival compared with conventional allogeneic transplant, along with open questions on cost, longer-term relapse control, and how the therapy compares with existing GVHD prevention approaches