Agios Pharmaceuticals Stock Soars After FDA Grants Priority Review for Mitapivat in Sickle Cell Disease

Regulation
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Summary · why it matters

Agios Pharmaceuticals shares surged 13.8% this week after the FDA granted priority review for its drug mitapivat as a treatment for sickle cell disease. Mitapivat, an oral pyruvate kinase activator already approved for PK deficiency and thalassemia, would be the first oral PK activator for sickle cell disease if approved. The priority review follows mixed Phase 3 RISE UP trial results, where mitapivat met hemoglobin response endpoints but failed to achieve statistical significance on the primary endpoint of reducing sickle cell pain crises. Agios later presented data suggesting a clinically meaningful benefit in pain crises among patients with a hemoglobin response. An accelerated approval decision is due by November 1, though full approval will require success in the confirmatory REIGNITE 3 trial, and competition looms from Novo Nordisk's rival PKR activator.

Impact on stocks 2

Biotech & Genomic Medicine± Mixed · 2 stocks
Agios Pharm
AGIO
▲ PositiveRegulationrelevance

FDA granted priority review for mitapivat in sickle cell disease, increasing approval odds.

Novo Nordisk A/S
NOV
▼ NegativeCompetitionrelevance

Novo Nordisk's rival PKR activator faces potential competition from mitapivat if approved.

Theme Impact 2

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