Novo submits etavopivat for FDA and EMA approval in sickle cell disease
- The FDA and EMA are expected to decide on the applications in 2027.
- Etavopivat is an investigational once-daily oral treatment for people aged 12 and older with sickle cell disease.
- Topline HIBISCUS results showed a 27% reduction in vaso-occlusive crises and an approximately four-month delay to the first crisis versus placebo.
- The HIBISCUS trial assessed crisis rates at week 52 and haemoglobin response at week 24; participants could continue standard-of-care treatment.
Regulatory submissions
Novo Nordisk 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, an investigational once-daily oral treatment for adolescents and adults aged 12 and older with sickle cell disease. Both agencies are expected to take action in 2027. The FDA has granted the medicine Fast Track, Rare Pediatric Disease and Orphan Drug designations; the European Commission has granted it Orphan Drug designation.
HIBISCUS results
The submissions are based on the HIBISCUS programme. In topline Phase 3 results announced in April 2026, etavopivat reduced vaso-occlusive crises by 27% and delayed the first crisis by approximately four months versus placebo. The 52-week trial assessed crisis rates at week 52 and haemoglobin response, defined as an increase of more than 1 g/dL, at week 24. Participants could continue standard-of-care treatment.
Mechanism and development
Etavopivat selectively activates pyruvate kinase red blood cell isozyme (PKR). The company says this reduces 2,3-diphosphoglycerate and increases ATP production, with the potential to reduce red blood cell sickling and improve cell survival. The programme also includes HIBISCUS 2, a Phase 3b study expected to enrol 510 people aged 12 and older, and FLORAL, an open-label extension collecting long-term safety data.
Source: Novo Nordisk