Vanda Pharmaceuticals (NASDAQ:VNDA) shares are up during Wednesday’s premarket session following the FDA’s approval of Bysanti, a new oral treatment for bipolar I disorder and schizophrenia, as well as the company’s rare skin disease drug is under FDA review.This approval marks a significant milestone for Vanda, as it positions the company to potentially capture a substantial market share in these therapeutic areas, which is reflected in the stock’s positive movement following the news.Seeks FDA Approval Fo ...

Faruqi & Faruqi, LLP Securities Litigation Partner James (Josh) Wilson Encourages Investors Who Suffered Significant Losses In Aquestive Therapeutics To Contact Him Directly To Discuss Their OptionsIf you suffered significant losses in Aquestive Therapeutics stock or options and would like to discuss your legal rights, call Faruqi & Faruqi partner Josh Wilson directly at 877-247-4292 or 212-983-9330 (Ext. 1310). [You may also click here for additional information]New York, New York--(Newsfile Corp. - Febru ...

Core Insights - Novartis (NVS) announced positive results from the late-stage III ALIGN study for its kidney disease drug Vanrafia (atrasentan) [1][9] - The ALIGN study demonstrated that Vanrafia effectively slows the decline of kidney function in patients with IgA nephropathy [3][4] Study Results - Vanrafia showed a significant difference in estimated glomerular filtration rate (eGFR) change from baseline compared to placebo, with a 2.39 ml/min/1.73m² difference at week 136 and a 2.59 ml/min/1.73m² difference at week 132 [3][4] - The study included a subgroup of patients receiving SGLT2 inhibitors, who also exhibited similar benefits [4] Regulatory Status - Vanrafia received accelerated approval in the United States and China in 2025 for reducing proteinuria in adults with IgA nephropathy, with plans for traditional approval in 2026 [5][9] Product Portfolio - Novartis' renal portfolio includes Fabhalta (iptacopan), which is approved for treating adults with paroxysmal nocturnal hemoglobinuria and has also received accelerated approval for IgA nephropathy [6] - Fabhalta generated sales of $155 million in Q4 and $505 million in 2025 [7] Market Context - 2026 is a critical year for Novartis due to the patent expiry of its cardiovascular drug Entresto, which is the largest in its history [8] - Novartis shares have increased by 52.9% over the past year, outperforming the industry growth of 20.6% [8]

Core Viewpoint - The U.S. Food and Drug Administration has declined to approve Aquestive Therapeutics' oral drug for severe allergic reactions, indicating a setback for the company in its product development efforts [1] Company Summary - Aquestive Therapeutics has faced a significant regulatory hurdle as the FDA did not approve its oral drug intended for treating severe allergic reactions, which may impact the company's future revenue and market position [1]

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]

Core Viewpoint - Shares of Travere Therapeutics (TVTX) declined by 14.6% following the FDA's announcement to extend the review timeline for the supplemental new drug application (sNDA) for Filspari in the FSGS indication by three months, with a new decision date set for April 13, 2026 [1][7]. FDA Review Extension - The FDA extended the review timeline after Travere submitted additional information to better define the clinical benefit of Filspari, classifying these submissions as a major amendment to the sNDA [2]. - No new safety or manufacturing data was requested by the FDA for Filspari [2]. Product Information - Filspari is currently approved to slow kidney function decline in adults with primary IgA nephropathy at risk of disease progression [3]. - FSGS is a rare kidney disorder affecting over 40,000 patients in the U.S. and a similar number in the EU, characterized by progressive kidney scarring and proteinuria [5][8]. - If approved for FSGS, Filspari would be the first FDA-approved therapy for this condition, addressing podocyte injury, a critical factor in FSGS progression [8]. Market Performance - Over the past six months, Travere Therapeutics' shares have increased by 72.1%, outperforming the industry average increase of 20.4% [4]. Financial Projections - Preliminary sales for Q4 2025 are expected to be around $127 million, with full-year 2025 net product sales projected at approximately $410 million in the U.S. [10]. - Preliminary U.S. net product sales of Filspari for Q4 2025 are estimated at $103 million, reflecting a 108% year-over-year increase, with full-year sales around $322 million [11]. Partnerships and Future Plans - In October 2025, Travere received a $40 million milestone payment from CSL Vifor after achieving market access milestones, with further payments expected tied to additional market access and sales [12]. - The company plans to continue building clinical evidence to support Filspari as a foundational treatment for IgAN through ongoing clinical studies, with the potential label expansion for FSGS likely to boost sales in 2026 [13].

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]

Group 1 - BofA raised the price target on Agios Pharmaceuticals (AGIO) to $34 from $32, maintaining a Buy rating on the shares [1] - The FDA approved mitapivat, making it the only drug approved for both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia [1] - The REMS and black box warning for hepatocellular injury were in line with expectations, and BofA does not anticipate this impacting drug uptake [1]