Novo Nordisk A/S (0QIU) announced topline results from HIBISCUS, a 52‑week pivotal phase 3 trial of once‑daily oral etavopivat in 385 people aged 12 and over with sickle cell disease. The trial met both co‑primary endpoints: etavopivat lowered the annualised rate of vaso‑occlusive crises (VOCs) by 27% versus placebo and extended median time to first VOC to 38.4 weeks from 20.9 weeks on placebo.
Haemoglobin response was markedly superior: 48.7% of treated patients achieved an Hb increase greater than 1g/dL at week 24 versus 7.2% with placebo, an adjusted rate difference of 41.2%. An exploratory analysis also showed a significant reduction in transfusion risk. Novo Nordisk said the topline safety profile was consistent with earlier studies.
Etavopivat, an oral PKR activator acquired with Forma Therapeutics in 2022, is being positioned as a disease‑modifying therapy. Novo Nordisk plans to submit for the first regulatory approval of etavopivat in the second half of 2026 and will present detailed HIBISCUS data at a scientific conference in 2026.
“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,” Martin Holst Lange said.
