While the FDA was originally expected to decide on the company’s application for Pyrukynd in treating thalassemia by September 7, its decision is now expected by December 7.
Agios Pharmaceuticals, Inc. (AGIO) announced on Thursday that the U.S. Food and Drug Administration has extended the review period for its drug Pyrukynd, intended for the treatment of adult patients with thalassemia, by three months.
Thalassemia is a rare, inherited blood disease that affects the production of the protein hemoglobin in red blood cells. By disrupting hemoglobin production, thalassemia reduces the number of circulating red blood cells and shortens their lifespan, resulting in anemia, fatigue, and severe complications.
While the FDA was initially expected to decide on the company’s application for Pyrukynd in treating thalassemia by September 7, its decision is now expected by December 7. On Stocktwits, retail sentiment around AGIO stock jumped from ‘bullish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘high’ to ‘extremely high’ levels.
A Stocktwits user opined that these are circumstances beyond the company’s control.
A pessimistic user, however, called for selling the company’s shares.
In the original application, the company had described the risk of hepatocellular injury with the drug. However, the firm stated on Thursday that it has submitted a proposed Risk Evaluation and Mitigation Strategy (REMS) in response to a recent information request from the agency.
The extension in date of the FDA’s decision is not the result of new or additional efficacy or safety data requested by the FDA or submitted by Agios, but a result of the new REMS submission, the company said. It further termed the REMS submission a major amendment to its original application.
“We remain confident in the favorable benefit-risk profile of PYRUKYND in thalassemia,” said CEO Brian Goff. As per Agios, approximately 6,000 adult patients are diagnosed with thalassemia in the U.S.
BofA subsequently lowered its price target on Agios Pharmaceuticals to $51 from $52 while keeping a ‘Buy’ rating on the shares. The firm noted that the extension was not the result of new data requested by the FDA or submitted by the company. BofA still views approval in thalassemia as likely given its key opinion leader checks and the strength of data from late-stage trials supporting the application. As such, Agios remains one of the firm’s top picks even with the delay, it said.
Pyrukynd is already approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. Pyruvate kinase (PK) deficiency is a rare, inherited disorder causing chronic hemolytic anemia in adults, where red blood cells break down too quickly due to a lack of the PK enzyme, resulting in low red blood cell count.
AGIO stock is up by 6% this year but down by over 22% over the past 12 months.
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