By Mackenzie Tatananni

Novo Nordisk reported encouraging data in a late-stage trial for etavopivat, a once-daily pill targeting sickle cell disease. The drugmaker's smaller rival is feeling the pressure.

Patients taking etavopivat along with their usual treatment saw a 27% reduction in the pain flares characteristic of the disease, Novo said. The drugmaker added that the treatment helped patients go four months longer before experiencing their first episodes, known in the medical community as a vaso-occlusive crisis event.

Etavopivat worked to manage anemia, too. Nearly half of the patients treated saw a significant improvement in their hemoglobin levels, compared with 7.2% of those who weren't on the medication. Hemoglobin is the protein in red blood cells that carries oxygen; when it's low, people feel tired and weak.

While Novo's American depositary receipts were unmoved by the news, shares of a smaller pharmaceutical company plunged.

With positive data in hand, Novo expects to apply for regulatory approval in the second half of the year. This is bad news for Agios Pharmaceuticals, whose development of a sickle-cell treatment has proven to be an uphill battle. Agios shares sank 24% on Monday.

Agios said earlier this month that it would pursue accelerated regulatory approval for its lead commercial product, mitapivat, following a March 31 meeting with the Food and Drug Administration. Mitapivat is already approved in patients with an inherited blood disorder called thalassemia.

It was a long time coming for Agios, which develops treatments for rare diseases. Shares tumbled in November 2025 after the company reported mixed results in a trial evaluating the efficacy of mitapivat in patients with sickle cell disease. Although the pill increased the concentration of hemoglobin in the blood, it failed to alleviate pain or fatigue.

Novo's progress toward securing a foothold in the market for sickle cell treatments threatens to overshadow any efforts by Agios to do the same. The current gold-standard treatment is an oral treatment called hydroxyurea, which is manufactured by major generic producers like Teva Pharmaceuticals.

Other drugmakers have attempted to make inroads in the market by targeting the root cause of the disease. Pfizer's voxelotor was seen as a first-in-class treatment to prevent blood cell sickling, but the company voluntarily withdrew all lots worldwide and discontinued active clinical trials.

The company determined that the drug's risks, namely an increased rate of pain events and death, outweighed its benefits.

Write to Mackenzie Tatananni at mackenzie.tatananni@barrons.com

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April 20, 2026 10:47 ET (14:47 GMT)

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