Key Points The new indication is relatively rare in the U.S., but pricing is substantial, and the need for the treatment is significant. The positive update turns the company's fortunes around after a disappointing update earlier in the year. 10 stocks we like better than Agios Pharmaceuticals › Shares in Agios Pharmaceuticals (NASDAQ: AGIO) soared by more than 18% as of 11 a.m. today. The move follows the company's receipt of approval from the Food and Drug Administration (FDA) for AQVESME (mitapiv ...

Core Insights - Agios Pharmaceuticals (AGIO) received FDA approval for Aqvesme (mitapivat) to treat adult patients with alpha- or beta-thalassemia, making it the only FDA-approved drug for both non-transfusion-dependent and transfusion-dependent forms of the disease [1][6] - The launch of Aqvesme is anticipated in late January 2026, following the implementation of the AQVESME REMS program [2][6] - The approval is based on phase III ENERGIZE and ENERGIZE-T studies, which showed significant improvements in hemoglobin levels, fatigue, and reduced blood transfusion needs compared to placebo [3][6] Regulatory and Market Context - The FDA's approval included a Risk Evaluation and Mitigation Strategy (REMS) due to reported cases of hepatocellular injury among treated patients, which may have negatively impacted investor sentiment, leading to a 1.5% decline in AGIO's stock [4][6] - AGIO's shares have decreased by 28.1% over the past year, while the industry has seen a rise of 16.8% [5] Future Developments - Agios has filed for regulatory approval of Aqvesme in the European Union, with a positive opinion from the EMA expected to lead to a final decision in early 2026 [9] - Outside the U.S., mitapivat will continue to be marketed as Pyrukynd for both PK deficiency and thalassemia indications, with ongoing studies for sickle cell disease [10]

To ensure this doesn’t happen in the future, please enable Javascript and cookies in your browser.If you have an ad-blocker enabled you may be blocked from proceeding. Please disable your ad-blocker and refresh. ...

U.S. stocks were higher, with the Dow Jones index gaining around 200 points on Wednesday.Shares of Dynavax Technologies Corp (NASDAQ:DVAX) rose sharply during Wednesday's session after Sanofi SA (NASDAQ:SNY) agreed to acquire the company for $15.50 per share in cash, reflecting a total equity value of approximately $2.2 billion.The acquisition augments Sanofi's presence in adult immunization.Dynavax shares jumped 38.6% to $15.44 on Wednesday.Here are some other big stocks recording gains in today’s session. ...

Shares of Agios Pharmaceuticals surged nearly 15% in premarket trading on Wednesday after the US Food and Drug Administration approved an expanded use of its drug mitapivat, strengthening investor opt... ...

Summary of Agios Pharmaceuticals Conference Call Company Overview - **Company**: Agios Pharmaceuticals (NasdaqGS:AGIO) - **Product**: Axazeni (mitapivat) for the treatment of anemia in adults with alpha or beta thalassemia Key Industry Insights - **FDA Approval**: Axazeni is the first and only medicine approved for both non-transfusion-dependent and transfusion-dependent alpha or beta thalassemia, regardless of transfusion burden [4][5] - **Market Opportunity**: Approximately 6,000 diagnosed adult thalassemia patients in the U.S., with an initial addressable population of about 4,000 patients [17][75] - **Pricing**: Axazeni is priced at approximately $425,000 per patient per year on a WAC basis, reflecting its differentiated benefits [22] Core Points and Arguments - **Clinical Significance**: Axazeni is the first disease-modifying treatment for non-transfusion-dependent thalassemia and has shown quality-of-life improvements in patients [5][11] - **Trial Results**: - In the ENERGIZE trial, 42.3% of patients achieved a hemoglobin response, with an average increase of 1.56 grams per deciliter [10] - In the ENERGIZE-T trial, 30.4% of transfusion-dependent patients met the primary endpoint of transfusion reduction response [11] - **Adverse Reactions**: Common adverse reactions included headache and insomnia, with a risk evaluation and mitigation strategy (REMS) in place for hepatocellular injury [12][15] Launch Strategy - **Commercial Model**: A capital-efficient global commercial model focusing on the U.S. market, which is the largest opportunity [16] - **Physician Engagement**: 86% of surveyed physicians plan to prescribe Axazeni within six months of availability, with strong familiarity with REMS programs [16] - **Initial Launch Dynamics**: Expectation of a 10-12 week period from prescription to treatment initiation due to REMS certification and prior authorization processes [63] Additional Considerations - **Long-term Growth**: The plan is to expand from the initial 4,000 addressable patients to the full 6,000 patient population over time, focusing on both transfusion-dependent and non-transfusion-dependent patients [75] - **International Launch**: Approval in KSA with expectations for slow initial demand generation in Gulf countries and Europe due to local regulatory processes [66][67] - **Future Developments**: Engagement with the FDA regarding the phase three RISE UP data for sickle cell disease and potential regulatory paths [23] Conclusion - The approval of Axazeni represents a significant advancement in thalassemia treatment, with a robust commercial strategy and strong clinical data supporting its launch. The company aims to address critical care gaps and establish Axazeni as the standard of care for thalassemia patients [85]

本文源自：格隆汇 美国制药商Agios Pharmaceuticals(AGIO.US)盘前涨近12%，报27.5美元。消息面上，Agios Pharmaceuticals表示，美国食品和药物管理局(FDA)已批准扩大其血液病药物的使用范围。该药名为 mitapivat，品牌名称为Aqvesme，目前正在接受测试，用于治疗α-或β-地中海贫血症成人患者的贫血 症。 ...

Core Viewpoint - Shares of Agios Pharmaceuticals experienced a nearly 12% increase in premarket trading following the approval from the U.S. Food and Drug Administration for the expanded use of its drug aimed at treating a specific type of blood disorder [1] Company Summary - Agios Pharmaceuticals received FDA approval for the expanded use of its drug, which is expected to enhance its market position and potentially increase revenue streams [1] - The approval is a significant milestone for Agios, indicating progress in its drug development and regulatory processes [1] Industry Summary - The approval of new treatments by the FDA can lead to increased competition within the pharmaceutical industry, particularly in the blood disorder treatment segment [1] - The market reaction, as evidenced by the share price increase, reflects investor confidence in the potential growth opportunities arising from regulatory approvals in the biotech sector [1]

美国制药商Agios Pharmaceuticals(AGIO.US)盘前涨近12%，报27.5美元。消息面上，Agios Pharmaceuticals表示，美国食品和药物管理局(FDA)已批准扩大其血液病药物的使用范围。该药名为 mitapivat，品牌名称为Aqvesme，目前正在接受测试，用于治疗α-或β-地中海贫血症成人患者的贫血 症。(格隆汇) ...

Group 1 - UiPath Inc. will join the S&P Midcap 400, replacing Synovus Financial Corp., effective January 2, 2026, leading to a 6.8% increase in shares to $17.05 in after-hours trading [1] - Agios Pharmaceuticals Inc. received FDA approval for AQVESME™ for treating anemia in adults with alpha- or beta-thalassemia, but shares fell 1.4% to $24.59 [1] - Can Fite Biopharma announced a 1-for-3,000 reverse share split, resulting in a 28.8% drop in shares to $0.17 in after-hours trading [1] - Citius Pharmaceuticals reported a quarterly loss of 11 cents per share, better than the expected loss of 43 cents, causing shares to surge 22.1% to $1.27 [1] - Ramaco Resources announced a $100 million stock repurchase plan, leading to a 7.1% increase in shares to $18.09 in after-hours trading [1]