Novo Nordisk’s Etavopivat Cracks Phase 3 Goals: A Potential New Standard of Care for Sickle Cell Disease

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Novo Nordisk’s Etavopivat hits the mark in Pivotal Sickle Cell Study. Etavopivat, an oral, once-daily pyruvate kinase-R (PKR) activator displayed supercilious results showing 27% reduction in the annualized rate of vaso-occlusive crises (VOCs) compared to placebo in 385 adolescents and adults on standard care.

Patients on etavopivat waited a median 38.4 weeks for their first VOC,nearly four months longer than the 20.9 weeks on placebo.Haemoglobin levels showed great improvement where 48.7% of etavopivat users saw a rise over 1 g/dL after 24 weeks, versus just 7.2% on placebo corresponding to an adjusted rate difference of 41.2%.

The trial hinted at fewer transfusions, with etavopivat appearing well tolerated.”Etavopivat has the potential to be a first and best-in-class therapy,” said Martin Holst Lange, Novo Nordisk’s EVP and Chief Scientific Officer. Novo Nordisk now plans to seek the first regulatory approval of etavopivat in the second half of 2026, with full trial results to be shared at a scientific conference later this year

By Pragna Biswas

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