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The new indication is relatively rare in the U.S., but pricing is substantial, and the need for the treatment is significant.
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The positive update turns the company’s fortunes around after a disappointing update earlier in the year.
Shares in Agios Pharmaceuticals (NASDAQ: AGIO) soared by more than 18% as of 11 a.m. today. The move follows the company’s receipt of approval from the Food and Drug Administration (FDA) for AQVESME (mitapivat) for the treatment of anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Additionally, management held a positive conference call to discuss the approval and outlined its marketing and pricing plans.
Thalassemia is an inherited blood disorder that leads to the body producing insufficient hemoglobin protein, causing excessive tiredness and weakness and ultimately increased morbidity. Mitapivat targets the mechanism within the red blood cells themselves, enabling stronger and longer-lasting red blood cells. As such, it is expected to reduce fatigue and the need for transfusions for sufferers.
During the conference call, the pharmaceutical company’s management noted it had an “addressable launch population” of 4,000 patients (Thalassemia is a relatively rare disease in the U.S., but more prevalent worldwide) and a price of $425,000 a year in the U.S.
Combined with its approval in other indications (mitapivat is already FDA approved for adults with Pyruvate Kinase Deficiency (PKD) and management believes it has the “potential to deliver $1bn global peak-year-sales across PKD and thalassemia indications.”That could increase if migratory trends toward the U.S. bring more potential thalassemia patients and their descendants into the U.S. market.
It’s a positive result after an earlier disappointment with mitapivat in a sickle cell disease trial, and the company is on track to rapidly expand sales following this approval.
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