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

• Sickle cell disease is an inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid, sticky, and sickle-shaped.
• The submissions are supported by results from the Phase 3 HIBISCUS trial, in which etavopivat demonstrated a 27% reduction in vaso-occlusive crises (VOCs) and an approximately four-month delay to the first VOC compared with placebo, based on topline results announced in April 2026.
• Novo Nordisk expects the FDA to take action on etavopivat's NDA in 2027, while the European MAA is also expected to receive a regulatory decision in 2027.

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.

What Did Phase 3 HIBISCUS Trial Show?

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. Participants were also allowed to continue receiving standard-of-care treatment during the trial.

Novo Nordisk said more detailed results from the Phase 3 study will be presented at a future scientific conference and published in a peer-reviewed journal.

What Is Etavopivat And How Does It Work?

Etavopivat is an investigational, once-daily oral selective activator of red blood cell pyruvate kinase (PKR), an enzyme involved in glycolysis.

According to Novo Nordisk, activating PKR is designed to reduce levels of 2,3-diphosphoglycerate (2,3-DPG) and increase adenosine triphosphate (ATP) production in red blood cells.

The reduction in 2,3-DPG is intended to improve hemoglobin's oxygen affinity and reduce sickle hemoglobin polymerization, while increased ATP production may help preserve red blood cell membrane integrity and survival.

When Could Etavopivat Receive Regulatory Decisions?

The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease, and Orphan Drug designations. The European Commission has also granted the drug Orphan Drug designation.

Novo Nordisk expects the FDA to take action on etavopivat's NDA in 2027, while the European Commission is expected to act on the MAA in 2027.

Etavopivat remains investigational and has not yet been approved to treat sickle cell disease.

NVO shares have declined approximately 20.1% year to date.

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