The U.S. Food and Drug Administration has extended its review timeline for Agios Pharmaceuticals’ (NASDAQ:AGIO) supplemental New Drug Application for PYRUKYND by three months, the company announced Thursday.
The new Prescription Drug User Fee Act goal date for the oral pyruvate kinase activator has been moved from September 7 to December 7, 2025. The extension follows Agios’ submission of a proposed Risk Evaluation and Mitigation Strategy to address potential hepatocellular injury risk.
The FDA classified this submission as a major amendment to the application, necessitating additional review time. Agios emphasized that the extension was not prompted by new safety or efficacy data.
"We remain confident in the favorable benefit-risk profile of PYRUKYND in thalassemia," said Brian Goff, Chief Executive Officer at Agios.
The application seeks approval for PYRUKYND to treat adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, a rare inherited blood disorder affecting approximately 6,000 adult patients in the U.S.
The submission is supported by data from two Phase 3 clinical trials - ENERGIZE and ENERGIZE-T - which evaluated the drug’s efficacy and safety in adults with thalassemia. The ENERGIZE trial focused on non-transfusion-dependent patients, while ENERGIZE-T studied transfusion-dependent individuals.
PYRUKYND is currently approved for treating hemolytic anemia in adults with pyruvate kinase deficiency, according to the company’s press release statement.
In other recent news, Agios Pharmaceuticals reported its Q2 2025 earnings, revealing a larger-than-expected loss per share, with an EPS of -$1.93, missing the forecast of -$1.81. However, the company achieved a notable revenue beat, with revenue surging to $12.5 million, surpassing expectations of $9.54 million. In response to safety concerns regarding its drug mitapivat, TD Cowen reiterated its Buy rating on Agios Pharmaceuticals, dismissing any link between the drug and reported adverse events. BofA Securities also maintained a Buy rating on the stock while raising its price target to $52 from $50. This adjustment followed the company’s clarification after reports of patient deaths appeared in the FDA’s adverse events reporting system. Additionally, Agios Pharmaceuticals addressed an analyst report concerning safety data for PYRUKYND, its treatment for hemolytic anemia. The report involved four patient cases, with three previously reported to the FDA as part of routine pharmacovigilance. These recent developments highlight the company’s ongoing efforts to manage safety concerns and meet financial expectations.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.