Etavopivat Shows Strong Phase III Results in Sickle Cell Disease

Novo Nordisk has reported positive outcomes from a Phase III trial of its once-daily oral therapy etavopivat for sickle cell disease (SCD), meeting both primary endpoints.

In the HIBISCUS study, the treatment reduced the annual rate of vaso-occlusive crises by 27% compared to placebo, while significantly extending the time to first crisis. The drug also improved haemoglobin levels, with nearly half of patients achieving a meaningful increase versus a small proportion in the placebo group.

Etavopivat, a pyruvate kinase-R activator, demonstrated a safety profile consistent with earlier trials. The study included 385 patients aged 12 years and above.

Following these findings, Novo Nordisk plans to seek regulatory approval in late 2026. The therapy could offer a more accessible alternative to high-cost gene treatments, especially as other pharmaceutical efforts in this space have faced setbacks.

20-04-2026