Novo Nordisk files etavopivat for sickle cell approval in US and Europe

Novo Nordisk has filed for approval of etavopivat on both sides of the Atlantic, handing a New Drug Application to the FDA and a Marketing Authorization Application to the European Medicines Agency. The once-daily pill is aimed at adolescents and adults aged 12 and older living with sickle cell disease. If cleared, it would be a disease-modifying option for a condition that still has few of them.

Both regulators are expected to act in 2027. Etavopivat already carries Fast Track, Rare Pediatric Disease and Orphan Drug designations from the FDA, plus Orphan Drug status from the European Commission. The filings rest on the Phase 3 HIBISCUS program, which measured hemoglobin response above 1 g/dL at week 24 and annualized vaso-occlusive crisis rates at week 52 against placebo.

The numbers behind the submission landed in April 2026, when topline results showed etavopivat cut vaso-occlusive crises by 27% and pushed back the first crisis by roughly four months compared to placebo. Participants stayed on standard of care throughout the trial. Novo says fuller HIBISCUS data will be presented at a future scientific conference and published in a peer-reviewed journal.

Etavopivat works by activating pyruvate kinase red blood cell isozyme, a key glycolysis enzyme. In sickle cell disease that activation lowers 2,3-DPG levels and boosts ATP production, which improves hemoglobin-oxygen affinity and helps keep red blood cells from sickling. The company says the effect preserves red blood cell membrane integrity and survival.

Martin Holst Lange, Novo's executive vice president, chief scientific officer and head of Research & Development, said severe vaso-occlusive crises and other symptoms have a profound impact on everyday life for people with the disease, and that treatment options remain limited. He said Novo hopes etavopivat can offer a new disease-modifying treatment and that the company will keep working with the community on health equity and the disparities tied to the disease.

Sickle cell disease is a progressive, life-threatening blood disorder driven by polymerization of mutated sickle hemoglobin. It affects an estimated 100,000 people in the United States, 110,000 in Europe and around 8 million worldwide, most of them in low and middle-income countries. Complications include acute chest syndrome, stroke, and kidney, liver and heart disease, and many patients see their lifespan cut by about 20 years. Novo picked up etavopivat through its 2022 acquisition of Forma Therapeutics. Beyond the pivotal HIBISCUS trial, the program includes the 510-patient Phase 3b HIBISCUS 2 study and FLORAL, an open-label extension collecting long-term safety data.