Novo Nordisk announced on April 21, 2026, that it will proceed with regulatory submissions for etavopivat, an investigational oral therapy for sickle cell disease, following successful results in the Phase 3 HIBISCUS clinical trial. The company confirmed plans to file for approval with the U.S. Food and Drug Administration and other global regulatory bodies during the second half of 2026. This development marks a significant milestone in the company’s expansion into rare blood disorders, a strategic pivot accelerated by its 2022 acquisition of Forma Therapeutics.
The HIBISCUS trial was a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of etavopivat in patients aged 12 to 65 with sickle cell disease. According to the data released by Novo Nordisk, the trial achieved its primary endpoint, demonstrating a statistically significant increase in hemoglobin levels of at least 1 g/dL from baseline at week 24 compared to the placebo group. Furthermore, the drug showed a favorable safety profile, consistent with earlier Phase 2 findings, with no new safety signals identified during the late-stage study.
Etavopivat functions as a selective erythrocyte pyruvate kinase activator. By increasing pyruvate kinase activity, the treatment aims to improve red blood cell metabolism, reduce 2,3-DPG levels, and increase ATP levels. This mechanism is intended to enhance hemoglobin oxygen affinity and improve the overall health and lifespan of red blood cells, thereby reducing the sickling process that leads to vaso-occlusive crises. In the HIBISCUS trial, secondary endpoints indicated a meaningful reduction in the annualized rate of vaso-occlusive crises, which are the leading cause of hospitalization for individuals living with the condition.
Martin Holst Lange, Executive Vice President of Development at Novo Nordisk, stated that the results reinforce the potential of etavopivat to serve as a foundational once-daily oral treatment. Lange noted that while recent advancements in gene therapy have provided new options for patients, there remains a substantial need for accessible, non-invasive therapies that can modify the course of the disease. The company’s 1.1 billion dollar acquisition of Forma Therapeutics in October 2022 was specifically targeted at securing etavopivat to bolster its hematology pipeline.
The upcoming regulatory filings will include comprehensive data from the HIBISCUS trial as well as long-term safety data from ongoing extension studies. Novo Nordisk indicated that it is currently finalizing the manufacturing and supply chain infrastructure required for a global launch, pending regulatory clearance. If approved, etavopivat would enter a competitive landscape that includes recently approved gene editing treatments and established therapies like hydroxyurea and voxelotor. The company has not yet provided specific pricing details for the therapy.