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Investing.com - Agios Pharmaceuticals (NASDAQ:AGIO), a biotech company currently trading at $35.28 with a market cap of $2.05 billion, has announced that the U.S. Food and Drug Administration (FDA) has extended the review period for its supplemental New Drug Application (sNDA) for mitapivat in thalassemia by three months. According to InvestingPro data, the company maintains a strong financial health score, with more cash than debt on its balance sheet.
The FDA requested that Agios submit a Risk Evaluation and Mitigation Strategy (REMS) protocol for the thalassemia indication, which the agency deemed a "major amendment" to the application. This extension moves the Prescription Drug User Fee Act (PDUFA) date from September to December 7, 2025. While the company’s current ratio of 14.48 indicates strong liquidity to manage through this extended timeline, InvestingPro analysis shows the company is quickly burning through cash.
The company indicated that the proposed REMS is specifically for thalassemia only, and the FDA has not requested any additional clinical or safety data beyond what was included in the original submission. Agios also noted that no new cases of drug-associated liver injury have been observed across the mitapivat program since the toxicity signal was reported at ASH 2024.
Agios currently uses a narrow distribution model for Pyrukynd (mitapivat) that provides patient and physician support, which could potentially be leveraged to capture liver enzyme test results if required by the final REMS protocol.
TD Cowen has maintained its Buy rating on Agios Pharmaceuticals following this announcement, viewing the extension rather than a Complete Response Letter (CRL) as a positive sign that approval will ultimately occur. While analyst targets suggest potential upside, InvestingPro subscribers have access to detailed Fair Value analysis and 10 additional ProTips that could provide crucial insights for investors considering this biotech stock. Get the complete picture with InvestingPro’s comprehensive research report, available for over 1,400 US stocks.
In other recent news, Agios Pharmaceuticals announced an extension of the Prescription Drug User Fee Act (PDUFA) date for its thalassemia treatment, PYRUKYND (mitapivat), from September 7 to December 7, 2025. This delay follows the company’s submission of a Risk Evaluation and Mitigation Strategy (REMS) to address potential hepatocellular injury, which the FDA considered a major amendment to the supplemental New Drug Application. Despite this extension, BofA Securities has adjusted its price target for Agios Pharma, lowering it from $52.00 to $51.00, while maintaining a Buy rating. Additionally, TD Cowen reiterated its Buy rating on Agios Pharmaceuticals, addressing recent safety concerns related to the drug mitapivat and stating that current reports do not support a link between the medication and patient deaths. Earlier, BofA Securities had raised its price target for Agios from $50.00 to $52.00, citing recent stock volatility. The company clarified that the PDUFA extension was not due to any new data requested by the FDA. These developments reflect ongoing regulatory and market dynamics surrounding Agios Pharmaceuticals.
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