CAMBRIDGE, Mass. - Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a $2 billion market cap biotechnology company, has announced the U.S. Food and Drug Administration's acceptance of its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) to treat adults with thalassemia, a rare blood disorder. The FDA has set a goal date of September 7, 2025, for its decision on this application. According to InvestingPro data, the company maintains strong financial health with more cash than debt on its balance sheet.
Thalassemia patients, who suffer from chronic anemia and risk severe complications, currently have limited treatment options. PYRUKYND, if approved, would offer a disease-modifying oral medication for both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia patients, regardless of genotype or transfusion needs. With revenue growth of nearly 37% in the last twelve months and a favorable P/E ratio of 2.95, Agios shows promising financial metrics despite being in the development stage.
The sNDA submission is based on findings from the ENERGIZE and ENERGIZE-T Phase 3 trials, which assessed the efficacy of mitapivat versus placebo. The ENERGIZE study focused on non-transfusion-dependent thalassemia and presented its results at the European Hematology Association 2024 Hybrid Congress in June 2024. The ENERGIZE-T study, targeting transfusion-dependent thalassemia, shared its outcomes at the American Society of Hematology Annual Meeting in December 2024.
Currently, PYRUKYND is approved in the U.S. for treating hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It is a first-in-class PK activator, and Agios is known for its work in cellular metabolism and rare hematologic diseases.
Safety information for PYRUKYND warns against abrupt discontinuation due to the risk of acute hemolysis and anemia. It also advises monitoring liver tests before and during treatment, as liver injury has been observed in patients treated with PYRUKYND at higher doses for other conditions. The most common adverse reactions include decreased estrone in males, increased urate, back pain, decreased estradiol in males, and arthralgia.
Agios' pipeline includes investigational medicines for other rare diseases, such as sickle cell disease and myelodysplastic syndrome (MDS)-associated anemia. The company's focus remains on providing transformative therapies for patients with rare diseases.
This announcement is based on a press release statement from Agios Pharmaceuticals, Inc. InvestingPro analysis indicates that analysts expect the company to remain profitable this year, with multiple additional ProTips available for subscribers. The company's next earnings report is scheduled for February 13, 2025, which could provide further insights into PYRUKYND's commercial progress.
In other recent news, Agios Pharmaceuticals has reported significant financial growth in its third quarter of 2024, driven by milestone payments amounting to $1.1 billion from the FDA approval of vorasidenib. The company also announced plans to launch its lead product, mitapivat, for thalassemia in 2025 and sickle cell disease in 2026. In addition, the FDA has approved an update to the prescribing information for Agios's drug PYRUKYND®, which includes new details on liver injury risks observed in patients treated with PYRUKYND for a condition different from its initially approved use.
In other developments, Agios has gained EU orphan drug status for its sickle cell treatment. The company's mitapivat is already approved in the United States and European Union as PYRUKYND® for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.
RBC Capital has increased its stock target for Agios from $55 to $57, maintaining an Outperform rating, following the presentation of promising clinical data. These recent developments highlight Agios Pharmaceuticals' ongoing efforts in the pharmaceutical sector.
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