Novo Nordisk’s etavopivat elicited a 27% drop in vaso-occlusive crises and 48.7% hemoglobin response after 24 weeks, creating “separation amongst PK class candidates,” Truist analysts said on Monday. Novo plans to seek FDA approval in the back half of 2026.
Novo Nordisk’s drug candidate for sickle cell disease significantly reduced severe pain crises in a Phase 3 study, opening the path for a regulatory submission later this year—and putting pressure on other drugmakers in this space.
The readout “creates separation amongst PK class candidates,” analysts at Truist Securities wrote to investors on Monday.
Data from the Phase 3 HIBISCUS study showed a 27% drop in vaso-occlusive crises (VOC) in patients taking etavopivat, as compared with placebo, according to Novo’s Monday morning release. The drug, taken orally once-daily, is a pyruvate kinase-R (PK) activator that works by preventing the sickling of red blood cells. Etavopivat came to Novo in September 2022 when the pharma acquired Forma Therapeutics for $1.1 billion.
Novo’s drug also delayed the time to the first VOC episode, with this occurring at 38.4 weeks in etavopivat-treated patients, compared to 20.9 weeks in placebo recipients. Hemoglobin response above 1 g/dL—a measure of how well a patient responded to the treatment—at 24 weeks reached 48.7% in the etavopivat arm versus 7.2% in placebo.
Novo will use the HIBISCUS readout to build toward a regulatory submission in the second half of 2026.
These data also shine a light on competitor programs. In its Monday note, Truist pointed to Agios Pharmaceuticals, which is working on its own orally available PK activator mitapivat for sickle cell disease (SCD).
Phase 3 data released in November 2025 demonstrated a 40.6% hemoglobin response in patients on mitapivat, compared with 2.9% in the placebo group. There have been no direct head-to-head studies assessing mitapivat against etavopivat, and cross-trial comparisons are inconclusive.
“We think AGIO could come under pressure,” Truist wrote, “and the competitor update is another threat to AGIO shares.” Still, the analysts noted that they still see “a place for mitapivat in SCD given the high unmet need globally.” Agios is down 15% in premarket trading on Monday.
Mitapivat is currently marketed under the brand name Pyrukynd for pyruvate kinase deficiency, for which it won approval in 2022. The drug picked up another approval for anemia in alpha- or beta-thalassemia in December.
Last month, Agios announced it was working with the FDA to settle on an accelerated pathway for mitapivat in SCD, with a supplemental drug application planned for “the coming months.”
If ultimately approved, Novo’s etavopivat will face off against other medicines in the sickle cell arena, including the gene therapies Casgevy (from Vertex Pharmaceuticals and CRISPR Therapeutics) and Lyfgenia (from bluebird bio). These therapies were approved in December 2023 but have had a hard time getting a foothold on the market.
At least for Casgevy, however, things seem to be turning around. Vertex and CRISPR said in February that patient infusions have been on the rise and that sales of the gene therapy could see encouraging growth this year.
