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Novo Nordisk’s sickle cell therapy hits in Phase 3, but data lag expectations

Novo Nordisk said Monday that its sickle cell disease pill succeeded in a late-stage study. However, the data came in behind expectations the company set last year.

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Novo Nordisk said Monday that its sickle cell disease pill succeeded in a late-stage study. However, the data came in behind expectations the company set last year.

Etavopivat cut the occurrence of the painful sickle cell complications known as vaso-occlusive crises (VOCs) by 27% compared with placebo, hitting one of the trial’s co-primary endpoints. However, the company’s then-head of rare disease Ludovic Helfgott told Endpoints News last year that Novo was hoping to see a 50% cut.

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