Key Takeaways
- Agios Pharmaceuticals shares fell [19%] after the FDA moved its mitapivat decision in sickle cell disease to Feb. 1, 2027.
- The bigger issue is whether the FDA requires a REMS safety program.
- Agios already sells mitapivat for thalassemia under a REMS and had $964.8 million of cash at the end of June.
- Novo Nordisk plans to file a drug that works the same way and faces the same safety question.
Agios Pharmaceuticals (AGIO) is down 18% today after the FDA pushed back its decision on mitapivat in sickle cell disease to Feb. 1, 2027.
That reaches Novo Nordisk (NVO) too: its etavopivat works the same way, and Novo plans to file it for sickle cell disease in the second half of 2026.
What happened
On Oct. 9, the FDA asked whether mitapivat needs a REMS in sickle cell disease. Agios sent about nine months of updated safety data on Oct. 19, and on Oct. 28 the FDA classified that response as a major amendment, adding three months.
Mitapivat has been sold for thalassemia as AQVESME since late January, only through a REMS, because of liver injury risk. Agios says no sickle cell patient on mitapivat has had liver injury with the pattern and severity seen in thalassemia, and that it’s in labeling talks with the FDA and “launch-ready.”
The label matters more
A REMS is the bigger worry: certified prescribers and paperwork for every new patient, in Agios’ biggest market yet.
And Agios knows the drill: mitapivat’s net revenue reached $44.7 million in the second quarter, up from $12.5 million a year earlier.
Analysts’ revenue estimates through 2028 include the sickle cell launch:
I’d expect the 2027 estimates to come down some, since they assumed a November launch.
Here’s what investors pay for those sales against Agios’ own history:
And Agios has the cash to wait:
It ended June with $964.8 million, after a second-quarter net loss of $100.7 million.
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Novo gets the same question
Novo’s etavopivat met both main goals of its Phase 3 trial in April, cutting pain crises by 27% against placebo, and Novo said its safety profile was in line with earlier trials.
Will the FDA ask Novo about a REMS too? Its results next week should say whether the filing is in.
My two cents, for what they’re worth: today’s drop is bigger than three months and a possible REMS deserve. A sickle cell REMS would slow the launch without stopping it.
Of course, the FDA could still ask for something stricter. Agios reports in early November: watch for any update on the labeling talks.
So what is Agios stock actually worth?
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