Welcome to our dedicated page for Agios Pharmaceuticals news (Ticker: AGIO), a resource for investors and traders seeking the latest updates and insights on Agios Pharmaceuticals stock.
Agios Pharmaceuticals (NASDAQ: AGIO) operates at the intersection of cellular metabolism science and rare genetic disease treatment. As a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, Agios generates news that matters to investors tracking the rare disease therapeutics sector.
The company's news flow reflects its dual identity as both a commercial organization with approved products and a research-driven enterprise advancing investigational therapies. Investors following AGIO stock can expect coverage of clinical trial results, regulatory decisions from agencies including the FDA and EMA, and commercial performance updates that directly impact the company's trajectory.
Agios's focus on rare hematologic diseases means their announcements often carry significant weight. Clinical data readouts for therapies targeting inherited blood disorders can move the stock substantially, as these conditions have limited treatment options and regulatory pathways frequently support accelerated approval. Partnership announcements with larger pharmaceutical companies also generate meaningful news, signaling external validation of the company's science and providing non-dilutive capital.
Beyond clinical milestones, Agios news includes quarterly earnings reports detailing product revenue, research expenses, and cash runway. For a company of this size, financial updates provide critical insight into commercial execution and the sustainability of ongoing research programs.
This page aggregates Agios Pharmaceuticals news from regulatory filings, press releases, and financial reports. Bookmark this resource to monitor developments affecting AGIO stock and the broader rare disease pharmaceutical landscape.
Agios (Nasdaq: AGIO) announced U.S. FDA approval of AQVESME™ (mitapivat) for treatment of anemia in adults with alpha- or beta-thalassemia, covering both non-transfusion-dependent and transfusion-dependent patients.
The approval is based on the randomized ENERGIZE and ENERGIZE-T Phase 3 trials (total 452 patients) that met primary and key secondary endpoints, showing improvements in hemoglobin, fatigue, and transfusion burden. AQVESME will be available in the U.S. in late January 2026 after implementation of an FDA REMS requiring liver tests and education. Five patients experienced adverse reactions suggestive of hepatocellular injury, with two hospitalizations; liver tests improved after discontinuation.
Agios (Nasdaq: AGIO) announced that the U.S. FDA has not issued a regulatory decision on the supplemental NDA for mitapivat in adult non‑transfusion‑dependent and transfusion‑dependent alpha‑ or beta‑thalassemia; the application remains under active review.
The FDA's PDUFA goal date was December 7, 2025. Agios said the agency has not requested, and the company has not submitted, any new efficacy or safety data, and that the companies are collaborating to finalize labeling and REMS materials. The FDA has not provided a timeline for its decision and Agios continues to work expeditiously to conclude the review.
Agios (Nasdaq: AGIO) reported topline 52-week RISE UP Phase 3 results for mitapivat in patients ≥16 with sickle cell disease. The trial met the primary hemoglobin response endpoint: 40.6% responders on mitapivat vs 2.9% on placebo (2-sided p<0.0001). Statistically significant improvements were also seen in average hemoglobin concentration (+7.69 g/L vs +0.26 g/L) and indirect bilirubin (-16.03 µmol/L vs +0.88 µmol/L) (both p<0.0001). The trial did not achieve statistical significance for annualized SCPC rate (2.62 vs 3.05, p=0.1213) or PROMIS Fatigue (p=0.7112). Safety was consistent with prior studies. Agios plans FDA engagement and intends an sNDA submission after a Q1 2026 pre-sNDA meeting.
Agios (Nasdaq: AGIO) will present new clinical and preclinical mitapivat data at the 67th ASH Annual Meeting, Dec 6–9, 2025 in Orlando.
Key highlights include a subgroup from ENERGIZE-T showing 77.8% (7/9) transfusion-reduction response in alpha-thalassemia versus 0% (0/3) placebo; long-term transfusion-free mean duration of 30.5 weeks (max 84.3 weeks) among 17 patients; and Phase 3 ACTIVATE-Kids meeting its primary hemoglobin endpoint with safety consistent with adult data. Ten Agios-led or collaborator presentations will be shared, plus advisory-council research on patient knowledge and fatigue impacts.
Agios (Nasdaq: AGIO) reported third quarter 2025 results and program updates on Oct 30, 2025. PYRUKYND net product revenue was $12.9M in Q3 2025, up 44% year‑over‑year. The FDA set a PDUFA goal date of Dec 7, 2025 for the PYRUKYND sNDA in thalassemia after requesting a REMS; CHMP adopted a positive opinion and an EC decision is expected by early 2026. Saudi Arabia approved PYRUKYND for thalassemia and commercial launch is underway there.
Topline results from the RISE UP Phase 3 sickle cell trial are expected by year‑end 2025, with a potential U.S. launch in 2026. The Phase 2b tebapivat LR‑MDS trial completed enrollment; topline data expected in early 2026. Cash, cash equivalents and marketable securities were $1.3B as of Sept 30, 2025.
Avanzanite Bioscience reports that partner Agios (NASDAQ: AGIO) received a positive CHMP opinion for PYRUKYND (mitapivat) as a treatment for anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.
Avanzanite entered an exclusive European commercialization agreement with Agios in June 2025 for PYRUKYND across the EEA, UK, and Switzerland. The CHMP opinion is based on Phase 3 ENERGIZE and ENERGIZE-T trial results. The European Commission will review the opinion, with a final decision expected by early 2026. PYRUKYND is already approved in Europe for adults with PK deficiency; thalassemia would be its second EU indication if authorized.
Agios (Nasdaq: AGIO) announced that the EMA CHMP adopted a positive opinion for PYRUKYND (mitapivat) to treat anemia in adults with transfusion‑dependent and non‑transfusion‑dependent alpha‑ or beta‑thalassemia, based on Phase 3 ENERGIZE‑T and ENERGIZE trials.
The European Commission will now review the opinion, with a final decision expected by early 2026. Agios signed an exclusive commercialization agreement with Avanzanite in June 2025 for the EEA, UK, and Switzerland and will support a potential European launch pending approval. PYRUKYND is approved in Saudi Arabia for thalassemia and has ongoing U.S. review with a PDUFA goal date of December 7, 2025. PYRUKYND is also approved for PK deficiency in the U.S. and Europe.
Agios (Nasdaq: AGIO) will report its third quarter 2025 financial results and business highlights on Thursday, October 30, 2025 at 8:00 a.m. ET.
The company will host a conference call and live webcast accessible from the Investors > Events & Presentations section of its website. A replay will be available on the company website approximately two hours after the event.
Agios Pharmaceuticals (Nasdaq: AGIO) announced that the FDA has extended the PDUFA goal date for PYRUKYND® (mitapivat) by three months, from September 7, 2025, to December 7, 2025. The extension follows Agios's submission of a proposed Risk Evaluation and Mitigation Strategy (REMS) to address hepatocellular injury risk.
The REMS submission is considered a major amendment to the supplemental New Drug Application (sNDA) for PYRUKYND®, which aims to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The extension is not related to new safety or efficacy data. The sNDA is supported by results from the ENERGIZE and ENERGIZE-T Phase 3 trials.
Agios Pharmaceuticals (Nasdaq: AGIO) has achieved a significant milestone as its drug PYRUKYND® (mitapivat) received approval from the Saudi Food and Drug Authority (SFDA) for treating adult patients with thalassemia. This marks the first regulatory approval for PYRUKYND in thalassemia globally.
The approval, evaluated under SFDA's Breakthrough Medicines Program, is based on results from the ENERGIZE and ENERGIZE-T Phase 3 trials. The drug is approved for both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia patients. Agios has partnered with NewBridge Pharmaceuticals to manage PYRUKYND commercialization in the Gulf Region.
Additional regulatory applications for PYRUKYND in thalassemia are under review in the U.S. (PDUFA date: September 7, 2025), United Arab Emirates, and European Union.
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