Novo Targets FDA Filing for Etavopivat After 27% VOC Reduction
Etavopivat cut vaso-occlusive crisis rates by 27% in a 385-patient phase 3 trial, and Novo Nordisk is now targeting an FDA submission in the second half of 2026 for the drug it acquired via the $1.1 billion Forma Therapeutics buyout.
The 52-week study enrolled patients aged 12 and older and hit both co-primary endpoints. Patients on the once-daily 400mg oral dose waited 38.4 weeks before their first VOC, compared to 20.9 weeks on placebo. At week 24, 48.7% of etavopivat patients showed a hemoglobin response of 1g/dL or greater, versus 7.2% on placebo: an adjusted rate difference of 41.2%. Novo also reported a safety profile consistent with earlier etavopivat trials.
If etavopivat clears FDA review, it enters a market where gene therapies Casgevy and Lyfgenia have grabbed early headlines but where an oral, once-daily option could reach patients who can’t access or afford cell-based treatments. That’s the scenario Novo paid $1.1 billion to own.
The competitive backdrop sharpens the stakes. Pfizer spent $5.4 billion acquiring Global Blood Therapeutics and got burned twice: one SCD asset failed phase 3, and Pfizer pulled the approved drug Oxbryta from markets worldwide in 2024. Novo’s CEO has already acknowledged a “difficult 2025.” A successful FDA submission would validate the $1.1 billion Forma price tag.
The phase 3 data says Novo has a shot. H2 2026 is when we find out if FDA agrees.
Sarah Chen
