The U.S. Food and Drug Administration (FDA) has approved an expanded indication for ACADIA Pharmaceuticals' blood disorder treatment drug, sending the company's stock surging nearly 12% in pre-market trading on Wednesday.
The company announced late Tuesday that its drug mitapivat has been approved for an additional indication, now covering the treatment of anemia in patients with non-transfusion-dependent or transfusion-dependent alpha- or beta-thalassemia.
Thalassemia is an inherited blood disorder that impairs the body's ability to produce hemoglobin and healthy red blood cells.
The newly approved drug, branded as Aqvesme, is expected to be commercially available by the end of January next year after completing required regulatory compliance procedures.
Notably, mitapivat was first approved by the FDA in 2022 under the brand name Pyrukynd for treating hemolytic anemia in adult patients with pyruvate kinase deficiency.
Gregory Lenzo, an analyst at Principal Financial Group, stated, "With this expanded indication, mitapivat could potentially add $320 million in peak revenue to its existing sales."
The FDA's approval was based on data from a Phase III clinical trial showing statistically significant improvement in hemoglobin response rates among patients treated with mitapivat compared to the placebo group.
Lenzo added that Aqvesme's prescribing information will include a black box warning: patients must undergo liver function tests every four weeks during the first 24 weeks of treatment, and the drug is contraindicated for patients with cirrhosis.
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