The results showed that etavopivat successfully met both co-primary endpoints, demonstrating superior reduction in vaso-occlusive crises (VOCs) and superior improvement in haemoglobin (Hb) response compared to placebo.

Etavopivat

Etavopivat is an oral, once-daily, pyruvate kinase-R (PKR) activator being developed to treat SCD, a seriously debilitating, life-threatening and life-shortening disease that impacts around 8 million people worldwide.

The HIBISCUS trial

The HIBISCUS trial was a randomised, double-blinded, 52-week efficacy and safety trial investigating etavopivat 400 mg versus placebo in 385 people aged 12 years or older with SCD. Participants were allowed to receive standard of care treatment throughout the trial.

In the trial, people treated with etavopivat demonstrated a superior reduction in the annualised rate of VOCs of 27% compared to placebo. The time to first VOC was significantly prolonged with etavopivat, with a median time to first VOC of 38.4 weeks versus 20.9 weeks for placebo.

In addition, etavopivat demonstrated a superior increase in the proportion of people achieving a Hb response greater than 1g/dL at week 24 of 48.7% compared to 7.2% with placebo, corresponding to an adjusted rate difference of 41.2%1. Further, as an exploratory analysis, etavopivat significantly reduced the risk of blood transfusion.

In the trial, etavopivat appeared to be well tolerated, with a topline safety profile in line with previous etavopivat trials.

We are very excited that etavopivat has the potential to be a first and best-in-class therapy and transform the lives of people with sickle cell disease, who currently have limited therapeutic options.

“Sickle cell disease severely impacts the lives of millions of people. We are very excited that etavopivat has the potential to be a first and best-in-class therapy and transform the lives of people with sickle cell disease, who currently have limited therapeutic options,” says Martin Holst Lange, executive vice president, chief scientific officer and head of Research and Development at Novo Nordisk. “Novo Nordisk remains committed to collaborating with sickle cell disease communities around the world to drive innovation, advance health equity and improve access to treatment and care.”

Novo Nordisk plans to submit for the first regulatory approval of etavopivat in the second half of 2026.