Shares in Agios Pharmaceuticals (AGIO +15.62%) were higher by more than 14% as of 11 a.m this morning. The move comes after a very positive development from the Food and Drug Administration (FDA) on its key drug, mitapivat.
Agios and mitapivat
The company already has mitapivat approved for adults with pyruvate kinase (PK) deficiency and for adults with alpha- or beta-thalassemia. However, it’s also seeking approval for sickle cell disease (SCD) based on a Phase 3 trial completed last year. The good news is the trial met its primary endpoint of “hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect bilirubin.”
Today’s Change
(15.62%) $5.84
Current Price
$43.23
Key Data Points
Market Cap
Day’s Range
$39.42 – $43.57
52wk Range
$22.24 – $46.00
Volume
77.8K
Avg Vol
1.1M
Gross Margin
82.07%
However, it did not meet the primary endpoint of demonstrating a statistically significant reduction in sickle cell pain crises (SCPCs). Sickle cells can build up in blood vessels, blocking blood flow and depriving organs of oxygen-rich blood, leading to acute pain.
What happened today
Despite failing to meet the SCPCs endpoint, Agios is seeking approval of mitapivat for SCD, and today the FDA announced it had accepted Agios’ supplemental New Drug Application (NDA) for mitapivat for SCD “with a Priority Review.” Moreover, it set a goal date for a decision by Nov.1.
Image source: Getty Images.
What it means for Agios Pharmaceuticals
This is a positive development that implies the FDA is sufficiently impressed by the data the pharmaceutical company submitted that it’s willing to grant it a faster review, despite mitapivat missing the SCPCs-related endpoint.
To be clear, though, this is for an accelerated approval. A full traditional approval is contingent on the REIGNITE Phase 3 Confirmatory Trial, which Agios is conducting under the FDA’s accelerated approval pathway, and those results won’t be available until next year at the earliest. Still, today’s news is a net positive, and investors are correct to welcome it.
Lee Samaha has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.
