Novo files sickle cell pill etavopivat with FDA and EMA

Novo Nordisk submitted etavopivat, its once daily oral sickle cell therapy, to the FDA and the EMA on November 13, on Phase 3 data showing a 27 percent reduction in painful vaso-occlusive crises.

Why it matters: Sickle cell disease has lost approved options in recent years, so a once daily oral disease modifying therapy with decisions expected in 2027 would reset the treatment landscape Novo bought into with its 1 billion dollar Forma Therapeutics deal.

Data as of Novo Nordisk submission announcement of November 13, 2026, read in full; Phase 3 HIBISCUS topline results announced April 2026. Regulatory action expected in 2027; neither agency has announced acceptance or a review date.

Novo Nordisk announced on November 13 that it has submitted a New Drug Application to the US Food and Drug Administration and a Marketing Authorization Application to the European Medicines Agency for etavopivat, a once daily, orally available selective activator of pyruvate kinase red blood cell isozyme, for adolescents and adults aged 12 years and older living with sickle cell disease. If approved, Novo positions etavopivat as a disease modifying therapy rather than a symptom treatment.

The filings rest on the HIBISCUS programme. The pivotal study enrolled approximately 440 people aged 12 and over who had experienced 2 to 15 vaso-occlusive crises in the prior 12 months, and measured the annualised rate of crises at week 52 and haemoglobin response at week 24 against placebo. In topline results announced in April 2026, etavopivat reduced vaso-occlusive crises by 27 percent and delayed the first crisis by about four months compared with placebo. Novo says fuller results will be presented at a future scientific conference and published in a peer reviewed journal, so the detailed dataset is not yet public. A second study, HIBISCUS 2, is designed to enrol 510 people.

The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations, and the European Commission has granted Orphan Drug designation. Novo expects the FDA to act on the application in 2027 and the European Commission to act on the European application in 2027. The company has not said that either application has been accepted for review, and no target action date has been announced.

Etavopivat came to Novo through the 2022 acquisition of Forma Therapeutics, a deal BioPharma Dive values at 1 billion dollars, as part of a deliberate build out in rare blood disorders beyond the company’s diabetes and obesity core. In a recent earnings presentation Novo described etavopivat, which is also in mid stage testing in thalassemia, as a future blockbuster and a candidate new standard of care in sickle cell disease. That commercial claim now depends on two 2027 regulatory decisions and on a full dataset that has yet to be shown.

Novo files sickle cell pill etavopivat with FDA and EMA

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