Novo Nordisk filed for approval of etavopivat in the US and Europe, the company announced on Friday. The drug is a once-daily oral treatment for sickle cell disease. Applications went to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The filings cover adolescents and adults aged 12 years and older. Both agencies are expected to decide in 2027.
The submissions rest on the Phase 3 HIBISCUS study. The trial enrolled roughly 440 patients aged 12 and older; topline data came out in April 2026. Etavopivat cut annualized vaso-occlusive crises by 27 percent versus placebo. It delayed the first crisis by approximately four months. Patients could stay on standard-of-care therapies during the 52-week trial. Etavopivat is a selective activator of pyruvate kinase red blood cell isozyme (PKR); it lowers 2,3-diphosphoglycerate levels and boosts adenosine triphosphate production to stop red blood cells from sickling. Novo Nordisk bought the compound in 2022 through its $1.1 billion purchase of Forma Therapeutics. The FDA granted Fast Track, Rare Pediatric Disease, and Orphan Drug status; European Orphan Drug designation also applies.
Sickle cell disease affects an estimated 100,000 Americans, 110,000 Europeans and roughly 8 million people worldwide, Novo Nordisk reported. Executive Vice President and Chief Scientific Officer Martin Holst Lange stated: "For people living with sickle cell disease, severe vaso-occlusive crises and other symptoms can have a profound impact on everyday life, and treatment options remain limited."
Novo Nordisk is running two companion studies: the 510-patient HIBISCUS 2 Phase 3b trial and the FLORAL open-label extension. Neither the FDA nor the EMA has reached a final approval decision. Voxelotor was withdrawn globally in 2024. Gene therapies Casgevy and Lyfgenia received regulatory approvals in December 2023; their high cost and myeloablative conditioning requirement restrict widespread use.
