Novo Nordisk Seeks FDA, EMA Approval For Sickle Cell Disease Treatment – Here Are The Key Takeaways

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Novo Nordisk said etavopivat could offer a new disease-modifying treatment option for people living with sickle cell disease if approved.

Novo Nordisk (NVO) said on Friday that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for etavopivat, an investigational once-daily oral treatment for adolescents and adults aged 12 and older living with sickle cell disease.

Sickle cell disease (SCD) is an inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid, sticky, and sickle-shaped. These cells can get stuck in small blood vessels and block blood flow, leading to severe pain episodes and other complications. Their premature breakdown can also cause anemia, while restricted blood flow can contribute to organ damage and stroke.

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"If approved, we are hopeful that etavopivat can provide a new disease-modifying treatment option for people living with sickle cell disease," said Martin Holst Lange, Novo Nordisk's executive vice president, chief scientific officer and head of Research & Development.

NVO shares traded marginally higher in premarket trading at the time of writing. However, retail sentiment on Stocktwits remained in the 'bearish' territory, amid 'normal message volumes in the past 24 hours.

The regulatory submissions are supported by data from the Phase 3 HIBISCUS trial program, which evaluated etavopivat's efficacy and safety against a placebo in adolescents and adults with sickle cell disease.

Topline results announced in April 2026 showed a 27% reduction in the annualized rate of vaso-occlusive crises (VOCs) and roughly a four-month delay to the first VOC compared with placebo, Novo Nordisk said.

The trial evaluated the annualized rate of VOCs at week 52 and the proportion of patients achieving a hemoglobin response of more than 1 g/dL at week 24.

Novo Nordisk said more detailed results from the Phase 3 study will…

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