Blood disease drugs were approved by the US FDA to expand the scope of use Agios Pharmaceuticals (AGIO.US) stock price soared by nearly 20%

Zhitong Finance App learned that the US Food and Drug Administration (FDA) approved the expanded indications of Agios Pharmaceuticals (AGIO.US) drugs to treat certain blood diseases, and the stock price rose sharply by 18.63% on Wednesday.

The company announced late Tuesday that its drug, whose chemical name is Mitapifal (trade name: Aqvesme), has been approved as the first oral treatment for patients with non-transfusion-dependent and transfusion-dependent alpha or beta thalassemia.

Thalassemia is an inherited blood disorder that affects the body's ability to produce hemoglobin and healthy red blood cells.

After implementing a required safety plan, Aqvesme is expected to go public later next month.

The drug will come with a black-box warning about hepatocyte damage and require liver function tests every four weeks during the first 24 weeks of treatment. The drug label will also advise patients with cirrhosis not to use it.

At least two analysts said that the drug label content and related requirements met expectations.

The company's chief financial officer, Cecilia Jones, said in an analyst conference call on Wednesday that the drug costs approximately $42.5 million per patient per year.

Eric Schmidt, an analyst at Cantor, said: “This is a slight premium over the $335,000 wholesale purchase cost price of Pyrukynd (another trade name for this drug). Our original expectation was affordable pricing.”

The drug (trade name: Pyrukynd) was approved by the FDA in 2022 to treat low red blood cell counts caused by pyruvate kinase deficiency in adults.

Gregory Renza, an analyst at Truist, said: “This approval unlocks an additional peak revenue opportunity of approximately $320 million on top of the existing Mitapivar product line.”

The company targets a group of about 6,000 adult thalassemia patients in the US. Of these, about 4,000 patients had access to treatment in the early stages of the drug's marketing.

The latest approval is based on a late-stage study in which patients treated with the drug showed a statistically significant improvement in hemoglobin response compared to the placebo group.