A Phase III trial of Novo Nordisk’s once-daily tablet has met both co-primary endpoints, reducing vaso-occlusive crises (VOC) and increasing haemoglobin (Hb) response in patients with sickle cell disease (SCD).
In the pivotal HIBISCUS trial (NCT04624659), the etavopivat arm demonstrated a 27% reduction in the annualised rate of VOCs compared to placebo, with time to first VOC being significantly prolonged with etavopivat at 38.4 weeks compared to 20.9 weeks for placebo.
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Etavopivat also demonstrated a 48.7% increase in the proportion of people achieving a Hb response greater than 1g/dL at week 24 compared to 7.2% with placebo.
In the trial, the pyruvate kinase-R (PKR) activator appeared to be well tolerated, with a topline safety profile in line with previous etavopivat trials.
“SCD 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.
Based on the study, which enrolled 385 SCD patients aged 12 and older, Novo Nordisk will submit for the first regulatory approval of etavopivat in H2 2026.
Novo Nordisk gained the rights to etavopivat in 2022 following the $1.1bn buyout of Forma.
According to GlobalData’s regional sales forecast, the drug is expected to bring in $174m in 2032. GlobalData is the parent company of Clinical Trials Arena.
This will be a significant success for Novo Nordisk, as Pfizer had also been eyeing up the SCD market with a $5.4bn buyout of Global Blood Therapeutics; however, the asset included in the deal failed to show significance in a Phase III trial. On top of that, in September 2024, Pfizer withdrew Oxbryta (voxelotor) in all approved markets after data suggested an imbalance in VOCs and fatal events.
In December 2023, the first gene therapies were approved for SCD. Casgevy, which was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, and bluebird bio’s Lyfgenia. Casgevy (exagamglogene autotemcel) brought in $116m in 2025, with 64 patients receiving the therapy.
The reason for a low patient uptake is likely the high costs of the therapies, at $2.2m for Casgevy and $3.1m for Lyfgenia, creating an access barrier for patients, meaning cheaper, alternative therapies will be beneficial.
