Blood disease drug approved by the FDA for expanded use, Agios Pharmaceuticals (AGIO.US) stock price soared nearly 20%.

The Food and Drug Administration (FDA) approved an expanded indication for Agios Pharmaceuticals (AGIO.US) drug to treat a certain blood disorder, causing the stock price to surge by 18.63% on Wednesday. The company announced on Tuesday evening that its drug, known chemically as mitapivat (brand name: Aqvesme), was approved, becoming the first oral therapy for non-transfusion dependent and transfusion-dependent or thalassemia patients. Thalassemia is a genetic 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 be on the market later next month. The drug will come with a black box warning for liver cell damage and will require liver function tests every four weeks during the first 24 weeks of treatment. The drug label will also advise against use in patients with liver cirrhosis. At least two analysts have expressed that the drug label content and related requirements are as expected. The company's CFO, Cecelia Jones, stated during a analyst call on Wednesday that the annual treatment cost per patient with the drug is approximately $425,000. Cantor analyst Eric Schmidt stated, "This represents a slight premium compared to the wholesale acquisition cost price of Pyrukynd (another brand name of the drug) of $335,000. We had originally anticipated pricing parity." The drug (brand name: Pyrukynd) was approved by the FDA in 2022 for the treatment of low red blood cell count in adults with pyruvate kinase deficiency. Truist analyst Gregory Lenza stated, "This approval builds on the existing mitapivat product line and unlocks approximately $320 million in peak revenue opportunities." The company targets approximately 6,000 adult thalassemia patients in the United States, with around 4,000 patients having the opportunity to receive treatment early on after the drug's market launch. This latest approval is based on a late-stage study in which patients treated with the drug showed statistically significant improvement in hemoglobin response compared to the placebo group.