Novo Nordisk seeks US and EU approval for sickle cell drug etavopivat

U.S. and European approval for a once-daily sickle cell pill is being sought as Novo Nordisk A/S CPHNOVO-B NYSENVO announced its etavopivat submissions on Friday. The applications follow a Phase 3 trial that met both main endpoints, including a 27 per cent reduction in painful vaso-occlusive crises versus placebo.

Novo's Nov. 13 announcement says it submitted a New Drug Application to the FDA and a Marketing Authorization Application to the European Medicines Agency for patients aged 12 and older. It expects regulatory action in 2027, but the announcement does not report acceptance for review or an exact decision deadline.

Novo is a large Denmark-based drugmaker that develops and markets medicines for chronic diseases worldwide.

Its U.S.-listed shares traded higher at US$40.73 at 9:37 a.m. EST on Nov. 13 in New York NYSENVO.

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The submissions move etavopivat from the company's clinical development programme into an application for commercial authorisation. They do not establish a launch date, approved label or reimbursement terms.

HIBISCUS Met Both Main Endpoints

Novo's April 20 filing described a randomised, double-blind trial in 385 participants comparing etavopivat 400 milligrams with placebo. Participants could continue standard care during the 52-week treatment period.

The co-primary measures were the annualised rate of vaso-occlusive crises and haemoglobin response. The latter meant an increase greater than one gram per decilitre at week 24.

Haemoglobin response occurred in 48.7 per cent of etavopivat recipients versus 7.2 per cent given placebo. Median time to the first crisis was 38.4 weeks with treatment, compared with 20.9 weeks on placebo.

Novo said etavopivat appeared well tolerated, with a topline safety profile consistent with previous trials. The filing's topline results section did not give p values, confidence intervals or adverse-event rates.

Friday's announcement said detailed Phase 3 results would be presented at a future scientific conference and published in a peer-reviewed journal. It did not identify a presentation date.

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"For people living with sickle cell disease, severe vaso-occlusive crises and other symptoms can have a profound impact on everyday life, and treatment options remain limited," Novo chief scientific officer Martin Holst Lange said in Friday's release.

Agios Provides A Competing Oral Approach

The closest mechanism-based comparator is Agios Pharmaceuticals, Inc. NASDAQAGIO, which reported its RISE UP results on Nov. 19, 2025. Its oral pyruvate kinase activator mitapivat met the haemoglobin-response endpoint but missed statistical significance on the annualised pain-crisis endpoint.

RISE UP enrolled 207 participants, with 138 receiving twice-daily mitapivat and 69 receiving placebo. Annualised pain-crisis rates were 2.62 and 3.05, respectively, with a p value of 0.1213.

Agios shares traded lower at US$31.23 at 9:35 a.m. EST on Nov. 13 in New York NASDAQAGIO.

Those separate trials provide no direct comparison between the drugs. Novo's results support its application with success on both prespecified main measures, while Agios' readout illustrates the difference between improving haemoglobin and demonstrating fewer crises.

The potential market remains underserved: Novo's Nov. 13 release estimates that sickle cell disease affects about 100,000 Americans and 110,000 Europeans. Etavopivat is designed to improve red blood cell energy production and reduce sickling through activation of the red blood cell form of pyruvate kinase.

Novo reports that the FDA previously granted Fast Track, Rare Pediatric Disease and Orphan Drug designations. Novo expects the FDA and European Commission to act on the applications in 2027. Its Nov. 13 release gives no calendar-day deadline for decisions on whether to authorise etavopivat.