Novo Nordisk Submits FDA New Drug Application And EMA Marketing Authorization Application For Etavopivat For Treatment Of Adolescents And Adults With Sickle Cell Disease
Benzinga Newsdesk 3 hours ago
FDANews
If approved, etavopivat could offer a new approach to treating SCD as a disease-modifying therapy. The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations and is
If approved, etavopivat could offer a new approach to treating SCD as a disease-modifying therapy. The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations and is expected to take action on the NDA in 2027. Etavopivat was also granted Orphan Drug designation by the European Commission who is expected to take action on the MAA in 2027. A MAA is the formal application for seeking approval to market a medicine in the European Union (EU).
The NDA and MAA submissions are grounded in the results from the HIBISCUS study program, which was designed to establish the efficacy and safety profile of etavopivat, and evaluated the annualized rate of vaso-occlusive crises (VOCs) at week 52 and hemoglobin response (>1 g/dL) rate at week 24 compared to placebo, in adults and adolescents living with SCD. Participants were also allowed to receive standard of care treatment throughout the trial.1 In topline results announced in April 2026, etavopivat showed a 27% reduction in VOCs and a ~4-month delay to the first VOC compared to placebo. More detailed results from the HIBISCUS phase 3 trial will be presented at a future scientific conference and published in a peer-reviewed journal.