Novo Nordisk Submits Sickle Cell Disease Treatment Etavopivat for US and European Regulatory Approval

Novo Nordisk (NYSE:NVO) has submitted regulatory applications in the United States and Europe seeking approval for etavopivat, an investigational oral treatment for sickle cell disease in patients aged 12 years and older.

The pharmaceutical company filed a New Drug Application (NDA) with the US Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA). Regulatory decisions on both applications are expected in 2027.

Etavopivat has previously received Fast Track, Rare Pediatric Disease and Orphan Drug designations from the FDA. The European Commission has also granted the treatment Orphan Drug designation.

These designations recognise specific aspects of the treatment’s development and the condition it is intended to address, but do not constitute regulatory approval.

Phase 3 HIBISCUS Results Support Applications

The submissions are supported by findings from the Phase 3 HIBISCUS clinical study programme, which evaluated etavopivat against placebo in adolescents and adults with sickle cell disease.

According to topline results announced in April 2026, treatment with etavopivat reduced vaso-occlusive crises by 27% compared with placebo.

The study also found that etavopivat delayed the first vaso-occlusive crisis by approximately four months relative to the placebo group.

Vaso-occlusive crises occur when sickled red blood cells obstruct blood flow, potentially causing episodes of pain and other complications.

Participants in the HIBISCUS programme were permitted to continue receiving standard-of-care treatment throughout the study, allowing etavopivat to be evaluated alongside existing disease management approaches.

The company expects to present the complete clinical findings at a future scientific conference and publish the results in a peer-reviewed medical journal.

Etavopivat Targets Red Blood Cell Function

Etavopivat is an investigational, once-daily oral treatment designed to selectively activate the red blood cell isozyme of pyruvate kinase, an enzyme involved in cellular energy metabolism.

The drug is being developed as a potential treatment for sickle cell disease, an inherited blood disorder characterised by abnormal haemoglobin and changes in the shape and function of red blood cells.

Sickle cell disease can result in recurrent vaso-occlusive crises and other complications associated with impaired blood flow.

Novo Nordisk is seeking approval for etavopivat in patients aged 12 and older, based on the population evaluated in its clinical development programme.

Martin Holst Lange, Executive Vice President and Chief Scientific Officer at Novo Nordisk, highlighted the need for additional therapies in a statement accompanying the announcement.

“Treatment options remain limited,” Lange said, referring to patients living with sickle cell disease.

Regulatory Reviews Expected to Continue Through 2027

The FDA and EMA will assess the submitted clinical and supporting data as part of their respective regulatory review processes.

Although the FDA has granted etavopivat several development-related designations, the treatment remains investigational and has not yet received marketing approval for sickle cell disease.

The anticipated regulatory decisions in 2027 represent the next major milestones for the programme.

Further information from the full HIBISCUS study results, including detailed efficacy and safety findings, is expected to become available through scientific presentation and peer-reviewed publication.

The applications expand Novo Nordisk’s regulatory pipeline beyond its established therapeutic areas, with etavopivat representing a potential additional treatment option for adolescents and adults with sickle cell disease, subject to regulatory approval.

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