股价飙涨近20%！美国FDA批准Agios Pharmaceuticals血液疾病药物扩大使用范围

Core Viewpoint - The FDA has approved Agios Pharmaceuticals' drug, mitapivat (brand name: Aqvesme), for the treatment of certain blood disorders, leading to an 18.63% increase in the company's stock price [1]. Group 1: Drug Approval and Market Impact - The drug Aqvesme is the first oral therapy approved for non-transfusion-dependent and transfusion-dependent alpha or beta thalassemia patients [3]. - The approval is based on a late-stage study showing statistically significant improvements in hemoglobin response compared to the placebo group [7]. - The company targets approximately 6,000 adult thalassemia patients in the U.S., with about 4,000 expected to have access to treatment at launch [7]. Group 2: Financial Aspects and Analyst Insights - The annual treatment cost for Aqvesme is approximately $425,000 per patient, which is a premium over the wholesale acquisition cost of $335,000 for another drug, Pyrukynd [6]. - Analysts estimate that the approval unlocks an additional peak revenue opportunity of about $320 million for the existing mitapivat product line [6]. - The drug will come with a black box warning regarding hepatocellular injury and will require liver function tests every four weeks during the first 24 weeks of treatment [5].