Agios Pharmaceuticals review

The short version

Agios was founded in 2008 to study how cells produce energy, and today it is built around one drug. Mitapivat is a pill that helps red blood cells work better, and it is already sold for two rare blood disorders. Revenue reached $44.7 million in Q2 2026, more than three times the level a year earlier (Agios Q2 2026 results). The big prize is sickle cell disease, which affects about 100,000 people in the US.

The latest FDA decision: delayed

On 29 October 2026, days before the 1 November deadline, Agios said the FDA had delayed its decision on sickle cell disease to 1 February 2027. The FDA had asked whether the drug needs a safety programme, Agios sent about nine months of new safety data, and the FDA called that a major addition, which adds three months by rule (HCPLive). Agios says it has seen no liver injury in sickle cell patients like the cases seen in thalassemia, and that talks on the label continue. The shares fell about 20% from the close before the news to the close after it, as our FDA Decision Calendar shows.

What it makes

Red blood cells need an enzyme called pyruvate kinase to produce energy. Mitapivat switches it on, which helps the cells last longer. It was first approved as Pyrukynd for pyruvate kinase deficiency. In January 2026 it launched in the US as Aqvesme for thalassemia, and thalassemia is also an approved use in the EU, Saudi Arabia and the UAE.

In the Phase 3 sickle cell trial, 40.6% of patients had a meaningful rise in hemoglobin vs 2.9% on placebo. Fewer patients needed a blood transfusion (23.9% vs 40.6%). But the rate of pain crises, the problem patients care about most, was 2.62 a year vs 3.05 on placebo, a difference too small to be statistically certain. That is why Agios is asking for an accelerated approval, which depends on a second trial to confirm the benefit.

Is it really new?

Yes. Mitapivat was the first drug of its kind, and no other approved medicine works this way. The idea is no longer unique, though: Novo Nordisk’s etavopivat uses the same mechanism and is in late trials. Agios itself dropped tebapivat, a second drug of this type, after disappointing results in sickle cell disease and a bone marrow disorder.

The competition

Sickle cell disease has more options than it did a few years ago, but none is simple and cheap. Gene therapies such as Vertex and CRISPR Therapeutics’ Casgevy cost over $2 million and need a long hospital stay. Pfizer’s pill Oxbryta was withdrawn in 2024 over safety concerns, which left a gap for a daily pill and also made regulators more careful. In thalassemia, Aqvesme competes with Bristol Myers Squibb’s injection Reblozyl.

Money

Agios lost $100.7 million in Q2 2026 and held $964.8 million of cash on 30 June, down from $1.16 billion at the end of 2025. That is enough for now, and the company says it covers a sickle cell launch. It also collects royalties from the cancer business it sold to Servier. Q3 results are due soon.

Risks

• Liver safety. Five of 301 thalassemia patients had liver injury reactions, two of them serious. A strict safety programme in sickle cell disease would slow sales.
• Weak result on pain crises. Doctors and insurers may hesitate if the drug raises hemoglobin but does not clearly prevent crises.
• Dependence on one drug. The pipeline behind mitapivat is early.
• Confirmatory trial. An accelerated approval can be withdrawn if the REIGNITE trial fails.

Our take

Agios has a real product and a real launch, and the sickle cell market could be several times larger than its current business. The delay did not change the data, but it put the spotlight on the drug’s main weakness. At a market value of about $1.9 billion, with almost $1 billion of cash on hand in June, the market is already pricing in a lot of doubt. The FDA’s answer in February will settle most of it.