Novo Nordisk is gearing up to submit its rare blood disorder drug to the FDA after the former Forma Therapeutics asset reduced a painful complication of sickle cell disease (SCD) in a phase 3 study.
The Danish pharma evaluated a once-daily, oral 400-mg dose of the pyruvate kinase-R (PKR) activator, called etavopivat, against placebo over 52 weeks in 385 people aged 12 years or older with SCD. The trial showed that people who received etavopivat experienced a 27% reduction in their annualized rate of vaso-occlusive crises (VOC) compared to the placebo cohort.
Patients on etavopivat also waited an average of 38.4 weeks before experiencing their first VOC compared to 20.9 weeks for the placebo group. VOCs occur in SCD patients when sickle-shaped red blood cells clog blood vessels, preventing oxygen from reaching tissues and causing severe pain in bones, the chest, the back or joints.