Core Viewpoint - Corcept Therapeutics faces significant setbacks regarding its proposed treatment for hypercortisolism, relacorilant, following warnings from the FDA not to submit its drug application, leading to a sharp decline in share price [1][2]. Company Developments - On January 30, 2026, Corcept shares dropped by $7.81, or 17%, after reports of FDA warnings against submitting the drug application [1]. - The company's market capitalization has decreased by $3.2 billion since December 30, 2025, following the receipt of a complete response letter (CRL) from the FDA [4]. FDA Communications - The FDA had previously issued a CRL indicating that it could not arrive at a favorable benefit-risk assessment for relacorilant without additional evidence of effectiveness [3]. - A corrected CRL dated January 28 revealed that the FDA had warned Corcept on multiple occasions about significant review issues if the application was submitted [3]. Investor Concerns - Hagens Berman, a law firm, is investigating whether Corcept misled investors regarding relacorilant's efficacy and the company's communications with the FDA [2][4]. - The firm is also analyzing if Corcept overstated its commercial and growth prospects for the hypercortisolism business, which was projected to grow from $3 billion to $5 billion in annual revenues within three to five years [3].

Core Viewpoint - Shares of Aquestive Therapeutics, Inc. (NASDAQ: AQST) dropped approximately 40% intraday following the FDA's identification of deficiencies in its New Drug Application (NDA) for Anaphylm, an experimental treatment for severe allergic reactions, including anaphylaxis [1] Group 1 - The FDA's findings prevent discussions regarding labeling and post-marketing requirements, raising concerns about the approvability of the application ahead of the January 31, 2026, PDUFA action date [1]

Key Takeaways TVTX shares fell after the FDA extended the Filspari sNDA review for FSGS by three months.The delay followed an amendment request, with no new safety or manufacturing data required by FDA.Filspari could become the first FDA-approved FSGS therapy, with Travere Therapeutics continuing launch prep.Shares of Travere Therapeutics (TVTX) were down 14.6% on Tuesday after the company announced that the FDA had extended the review timeline for the supplemental new drug application (sNDA) seeking label ...

Core Insights - Corcept Therapeutics faced a significant setback as the FDA issued a complete response letter, declining to approve its relacorilant medication, leading to a share price drop of over 50% [1][2][4] - The FDA acknowledged that relacorilant met its primary endpoint in clinical trials but required additional evidence of effectiveness for a favorable benefit-risk assessment [3][4] - Corcept plans to continue pursuing commercialization of relacorilant and intends to meet with FDA officials to discuss potential next steps [3][4] Company Performance - The company's share price experienced a drastic decline of just over 50% following the FDA's decision [1] - The FDA's rejection is viewed as a significant defeat for Corcept, indicating challenges ahead for the drug's approval process [4][7] Future Prospects - Corcept is determined to find a way to commercialize relacorilant, although this may involve additional clinical trials, complicating the approval process [3][4] - The FDA's response suggests that any future success will require substantial evidence to support the drug's effectiveness [4][7]

Core Viewpoint - Agios Pharmaceuticals received FDA approval for mitapivat (AQVESME), leading to an 18% increase in share price and a price target raise by BofA Securities from $32 to $34 while maintaining a Buy rating [1] Group 1: FDA Approval and Market Impact - Mitapivat is now the only approved therapy for both transfusion-dependent and non-transfusion-dependent alpha- and beta-thalassemia [2] - Despite delays around the PDUFA date, confidence in the approval was high due to prior regulatory clearance in Saudi Arabia and a favorable opinion from the European Medicines Agency's CHMP [2] Group 2: Drug Launch and Sales Projections - The drug's REMS requirements and black box warning related to hepatocellular injury are consistent with expectations and are not anticipated to significantly hinder adoption [3] - The launch execution for thalassemia is expected to be a major focus for 2026, with availability anticipated in late January 2026 [3] - BofA projected peak sales of approximately $1 billion for the thalassemia indication, with initial uptake expected to be gradual due to REMS certification requirements [4] Group 3: Probability of Success and Market Share - Following the approval, BofA raised the probability of success in thalassemia to 100% from 85% and modestly increased peak market share assumptions [4]

BofA raised the firm’s price target on Agios Pharmaceuticals (AGIO) to $34 from $32 and keeps a Buy rating on the shares following FDA approval for mitapivat to make it the only drug approved in both transfusion-dependent and non-transfusion-dependent alpha- or beta- thalassemia. The REMS and black box for hepatocellular injury were in line with the firm’s expectations and BofA doesn’t expect this to impact uptake, the analyst tells investors. Claim 70% Off TipRanks This Holiday Season Published first on ...

Group 1 - Omeros (OMER) stock experienced a significant surge following the FDA approval of its drug candidate narsoplimab for treating hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) [1] - The drug will be marketed and sold under a specific brand name, although the name is not mentioned in the article [1] - The article highlights the importance of staying updated on stocks within the biotech, pharma, and healthcare industries, suggesting a subscription to a weekly newsletter for insights on key trends and catalysts [1] Group 2 - Edmund Ingham, a biotech consultant, has over five years of experience covering the biotech, healthcare, and pharma sectors, and has compiled detailed reports on more than 1,000 companies [1] - The investing group Haggerston BioHealth provides resources for both novice and experienced biotech investors, including catalysts to monitor, buy and sell ratings, product sales forecasts, and integrated financial statements [1] - The group also conducts discounted cash flow analysis and market-by-market analysis for major pharmaceutical companies [1]

Core Viewpoint - Shares of Agios Pharmaceuticals increased nearly 15% in premarket trading following the FDA's approval for an expanded use of its drug mitapivat, indicating strong investor optimism and potential growth for the company [1] Company Summary - Agios Pharmaceuticals received FDA approval for an expanded indication of mitapivat, which is expected to enhance the drug's market potential and drive revenue growth [1] - The approval is likely to strengthen investor confidence in Agios Pharmaceuticals, as reflected in the significant premarket share price increase [1]

Group 1 - The U.S. Food and Drug Administration has approved GSK's oral treatment for gonorrhea, a sexually transmitted infection [1]

Core Insights - Agios Pharmaceuticals (AGIO) is awaiting a decision from the FDA regarding its supplemental new drug application (sNDA) for Pyrukynd (mitapivat) to treat thalassemia, which has missed the expected deadline of December 7, 2025 [1][2][7] - The sNDA is under active review, and no additional efficacy or safety data have been requested by the FDA [2][8] - Pyrukynd is already approved in the U.S. and Europe for treating hemolytic anemia in adults with pyruvate kinase deficiency [3][7] Company Performance - Year-to-date, Agios shares have declined by 17%, contrasting with a 19.3% increase in the industry [4] - The FDA extended the review timeline for the sNDA by three months in September 2025 due to a request for a Risk Evaluation and Mitigation Strategy (REMS) related to potential liver injury risks [5] Regulatory Developments - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Pyrukynd's label expansion in thalassemia, with a final decision from the European Commission expected in early 2026 [8] - Besides thalassemia, Pyrukynd is also being studied for sickle cell disease (SCD), where recent phase III trial results were mixed, meeting one primary endpoint but failing another [9]