Global pharma giant Novo Nordisk has unveiled headline results from its HIBISCUS phase 3 trial of once-daily oral etavopivat. This marks a potential breakthrough for sickle cell disease (SCD), a severe and life-limiting condition affecting around 8 million people worldwide.

 

The study delivered a clear win on both co-primary endpoints. Etavopivat showed a superior reduction in vaso-occlusive crises (VOCs) compared with placebo, alongside a significantly stronger improvement in haemoglobin response.

 

Etavopivat is an oral, once-daily pyruvate kinase-R (PKR) activator designed to address the underlying biology of SCD, a disease that causes recurrent pain episodes, organ damage and reduced life expectancy.

 

The 52-week, randomised, double-blind trial enrolled 385 adults and adolescents aged 12 and older, all of whom continued on standard of care treatments throughout.

 

The results were clinically meaningful. Patients receiving etavopivat experienced a 27% reduction in the annualised rate of VOCs versus placebo. Time to first crisis was also extended, with a median of 38.4 weeks compared with 20.9 weeks for placebo.

 

“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,” said 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.”