BAGSVAERD, DENMARK — Novo Nordisk today announced positive topline results from HIBISCUS, a pivotal Phase 3 trial evaluating etavopivat, a once-daily oral PKR activator for adults and adolescents living with sickle cell disease (SCD). The trial successfully met both co-primary endpoints, marking a significant milestone for a disease that affects approximately 8 million people globally.
Superior Clinical Outcomes The HIBISCUS trial, which included 385 participants aged 12 and older, demonstrated that etavopivat significantly reduces the burden of the disease. Key findings include:
- VOC Reduction: Patients treated with etavopivat saw a 27% reduction in the annualized rate of vaso-occlusive crises (VOCs) compared to those on a placebo.
- Delayed Crisis Events: The median time to the first VOC was nearly doubled, extending to 38.4 weeks with etavopivat compared to 20.9 weeks for the placebo group.
- Hemoglobin Response: An impressive 48.7% of participants achieved a hemoglobin increase of more than 1g/dL after 24 weeks, a stark contrast to the 7.2% observed in the placebo group.
A New Class of Therapy Etavopivat works as a pyruvate kinase-R (PKR) activator. By improving red blood cell health and oxygen affinity, it addresses the underlying causes of SCD rather than just the symptoms. Beyond the primary endpoints, exploratory analysis also showed a significant reduction in the risk of blood transfusions, further highlighting the drug’s potential to improve patient quality of life.
“We are very excited that etavopivat has the potential to be a first and best-in-class therapy,” said Martin Holst Lange, Executive Vice President and Head of R&D at Novo Nordisk. “This treatment could transform the lives of people with sickle cell disease who currently face very limited therapeutic options.”
Regulatory Timeline The safety profile of etavopivat remained consistent with previous trials, appearing well-tolerated throughout the 52-week study. Novo Nordisk has confirmed plans to submit the drug for its first regulatory approval in the second half of 2026. Detailed results from the HIBISCUS trial are expected to be presented at major international scientific conferences later this year.
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