Agios Pharmaceuticals Inc (NASDAQ:AGIO) shares are trading sharply higher Tuesday afternoon after the company said it will pursue U.S. accelerated approval for mitapivat in sickle cell disease following a pre-supplemental New Drug Application meeting with the FDA.
- Agios Pharmaceuticals stock is charging ahead with explosive momentum. What’s driving AGIO stock higher?
Accelerated Approval Path Gives Agios A Clearer Regulatory Catalyst
The update appears to be boosting investor sentiment because it marks a potentially faster regulatory path for mitapivat, an oral pyruvate kinase activator that Agios is developing for sickle cell disease.
According to the company, the FDA recommended submission of a confirmatory clinical trial proposal to support accelerated approval, and Agios said it has already submitted that proposal for review. The company also plans to submit the supplemental NDA in the coming months.
Phase 3 Hemoglobin Response Supports Mitapivat's Sickle Cell Case
Agios said the regulatory discussions were based on data from its RISE UP clinical program, including Phase 2 and Phase 3 studies. In the Phase 3 trial, mitapivat achieved a statistically significant improvement in hemoglobin response compared with placebo, although the annualized rate of sickle cell pain crises did not reach statistical significance.
Still, the company said patients who met the hemoglobin response endpoint also showed clinically meaningful improvements in fatigue, reductions in pain crises and fewer related hospital visits.
AGIO Shares Surge Tuesday Afternoon
AGIO Price Action: Agios Pharmaceuticals shares were up 20.16% at $35.58 at the time of publication on Tuesday, according to Benzinga Pro data.
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