Agios Pharma Shares Surge 16% on FDA Drug Approval for Thalassemia Treatment
Agios Pharmaceuticals' stock jumped 16% after FDA approved expanded use of mitapivat (Aqvesme) for treating anemia in alpha- or beta-thalassemia patients. The drug, priced at $425,000 per patient annually, is the first oral treatment for this condition. It targets 6,000 adult thalassemia patients in the US, with 4,000 addressable at launch. The approval could add $320 million in peak revenue opportunity.
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Agios Pharmaceuticals witnessed a remarkable stock performance on Wednesday, with shares jumping nearly 16% following a significant regulatory milestone. The surge came after the US Food and Drug Administration (FDA) approved the expanded use of the company's drug for treating a specific type of blood disorder.
FDA Approval Details
The drug mitapivat, marketed under the brand name Aqvesme, received approval as the first oral treatment for patients with anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The company announced this development late on Tuesday, marking a significant expansion of the drug's therapeutic applications.
| Parameter | Details |
|---|---|
| Drug Name | Mitapivat (Aqvesme) |
| Treatment Type | First oral treatment for thalassemia-related anemia |
| Patient Categories | Non-transfusion-dependent and transfusion-dependent |
| Availability | Expected late next month |
Thalassemia is an inherited blood disorder that affects the body's ability to produce hemoglobin and healthy red blood cells, making this approval particularly significant for patients suffering from this condition.
Safety Requirements and Pricing
Aqvesme will be available following the implementation of a required safety program. The drug carries a boxed warning for hepatocellular injury and mandates liver function tests every four weeks during the first 24 weeks of treatment. Additionally, the label advises against use in patients with cirrhosis.
Chief Financial Officer Cecilia Jones revealed during an analyst call on Wednesday that the yearly price of the drug is approximately $425,000 per patient. This pricing represents a modest premium compared to Pyrukynd's wholesale acquisition cost price of $335,000, according to Cantor analyst Eric Schmidt.
Market Opportunity and Revenue Projections
The approval significantly expands Agios Pharmaceuticals' market reach. The FDA previously approved the drug in 2022 under the brand name Pyrukynd for treating low red blood cell counts in adults with pyruvate kinase deficiency.
| Market Metrics | Numbers |
|---|---|
| Target US Population | 6,000 adult thalassemia patients |
| Addressable at Launch | 4,000 patients |
| Additional Peak Revenue Opportunity | $320 million |
Truist analyst Gregory Renza highlighted that "the approval unlocks an additional $320 million in peak revenue opportunity layered atop the existing mitapivat franchise." The company targets a population of approximately 6,000 adult thalassemia patients in the US, with about 4,000 expected to be addressable at launch.
Clinical Efficacy
The latest approval is supported by robust clinical data from a late-stage study. Patients receiving the drug demonstrated a statistically significant increase in hemoglobin response compared with placebo, providing strong evidence for the treatment's effectiveness. At least two analysts indicated that the label requirements and safety protocols were in line with expectations, suggesting the approval process proceeded as anticipated by market observers.
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