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 the tissues and causing severe pain in the bones, chest, back or joints.
Meanwhile, 48.7% of patients who received etavopivat showed a haemoglobin response of 1g/dL or greater at week 24, compared to 7.2% of those on placebo. This equated to an adjusted rate difference of 41.2%, Novo explained in an April 20 release.
The VOC and hemoglobin data mean the study hit both of its co-primary endpoints. The drug also appeared to be well tolerated, according to Novo, with a “topline safety profile in line with previous etavopivat trials.”
It means the pharma is now gearing up to submit an approval application for etavopivat in the second half of the year.
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Etavopivat was at the center of Novo’s decision to acquire Forma for $1.1 billion back in 2022. At the time, Novo’s hope was that the acquisition could help build a leading portfolio in SCD.
“Sickle cell disease severely impacts the lives of millions of people,” Novo’s Chief Scientific Officer Martin Holst Lange, M.D., Ph.D., said in this morning’s release. “We are very excited that etavopivat has the potential to be a first and best-in-class therapy and transform the lives of people with sickle cell disease, who currently have limited therapeutic options.”
While even Novo CEO Maziar Mike Doustdar has previously acknowledged the Wegovy maker had a “difficult 2025,” today’s phase 3 win is a reminder that the company’s pipeline still has promise.
And while Novo has reason to hope that its SCD bet will pay off with an approval, some of its Big Pharma peers have struggled. Pfizer spent $5.4 billion to purchase Global Blood Therapeutics, but not only did one SCD asset from that buyout fail a key phase 3 test, but Pfizer also had to pull GBT’s approved SCD drug Oxbryta from markets worldwide in 2024.
The biggest positive developments in the SCD space in recent years has been from gene therapies, notably Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy as well as bluebird bio’s Lyfgenia.
