The U.S. Food and Drug Administration on Thursday approved GSK's oral treatment for gonorrhea, a sexually transmitted infection. ...

Key Takeaways AGIO says the FDA missed its Dec. 7 deadline for a Pyrukynd thalassemia sNDA decision.The sNDA remains under FDA review but no additional efficacy or safety data have been requested.Pyrukynd is already approved for treating hemolytic anemia in adults with PK deficiency.Agios Pharmaceuticals (AGIO) announced that the FDA has not yet given its decision related to the company’s supplemental new drug application (sNDA) seeking approval for its only marketed drug, Pyrukynd (mitapivat), for treating ...

Regulatory Overview - The FDA has approved 34 novel drugs from January to October 2025, compared to 38 during the same period in 2024 [2] - Several biotech stocks are highlighted as they approach key regulatory decisions in November 2025 [2] Arrowhead Pharmaceuticals Inc. (ARWR) - ARWR's New Drug Application for Plozasiran is under FDA review, with a decision expected on November 18, 2025 [3] - Plozasiran targets familial chylomicronemia syndrome, a condition characterized by extremely high triglyceride levels, which can lead to acute pancreatitis [3] - Clinical trials showed significant reductions in triglycerides and the incidence of acute pancreatitis, potentially providing a new treatment option alongside Tryngolza, the only FDA-approved drug for this condition [4] Otsuka Holdings Co. Ltd. (OTSKF) - OTSKF is awaiting the FDA's decision on Sibeprenlimab, scheduled for November 28, 2025 [6] - Sibeprenlimab is an investigational monoclonal antibody for treating immunoglobulin A nephropathy (IgAN), a chronic kidney disease [6] - Clinical trials demonstrated a significant reduction in urine protein-to-creatinine ratio, indicating its potential as a blockbuster drug if approved [8] Bayer (BAYRY) - Bayer's investigational compound Sevabertinib is under priority review by the FDA, with a decision expected on November 28, 2025 [9] - Sevabertinib is proposed for treating advanced non-small cell lung cancer (NSCLC) with HER2 mutations, providing an additional option for previously treated patients [10] Ascendis Pharma A/S (ASND) - The FDA decision on Navepegritide, aimed at treating children with achondroplasia, is due on November 30, 2025 [11] - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to serious complications [12] - Navepegritide is designed for weekly subcutaneous administration, targeting the overactive FGFR3 pathway [13] Kura Oncology Inc. (KURA) - KURA is expecting FDA approval for Ziftomenib on November 30, 2025, for treating relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations [15] - Ziftomenib has shown significant efficacy in clinical trials, with a market opportunity estimated at $350 million to $400 million annually in the U.S. if approved [17]

Core Insights - Corcept Therapeutics' sole-marketed drug, Korlym, has shown strong sales performance, with $351.6 million in sales for the first half of 2025, reflecting a year-over-year increase of approximately 13.2% [1][9] - The company anticipates full-year 2025 Korlym sales to reach $857.1 million, indicating a nearly 27% increase year-over-year [1][3] - Management expects a significant rise in Korlym prescriptions in the second half of 2025 as vendor issues are resolved [2][3] Sales and Revenue Expectations - Korlym sales were impacted by vendor capacity issues in Q1 2025 but improved in Q2, with expectations for continued growth in H2 2025 [2][3] - Corcept projects total revenues for 2025 to be between $850 million and $900 million [3] Pipeline Developments - Corcept is developing relacorilant for Cushing's syndrome, with a new drug application submitted to the FDA, and a decision expected by December 30, 2025 [4][9] - Approval of relacorilant is anticipated to broaden the patient base and reduce reliance on Korlym for future growth [5] Additional Indications and Studies - The company is also pursuing relacorilant for other indications, including a combination therapy for platinum-resistant ovarian cancer and adrenal cancer [6][7] - The BELLA study is evaluating the efficacy of relacorilant combined with nab-paclitaxel and Avastin for ovarian cancer treatment [7] Market Performance and Valuation - Corcept's stock has increased by 38.3% year-to-date, outperforming the industry average of 8.3% [8] - The company's shares are trading at a premium, with a price-to-sales ratio of 11.73 compared to the industry average of 2.38 [10] Earnings Estimates - The Zacks Consensus Estimate for 2025 earnings per share has decreased from $1.39 to $1.15, and for 2026, it has decreased from $2.08 to $1.71 [11]

Core Viewpoint - Outlook Therapeutics' stock experienced a significant decline following the FDA's issuance of a complete response letter (CRL) regarding its biologics license application (BLA) for ONS-5010, a treatment for wet age-related macular degeneration (wet AMD) [1][2][9] FDA Update - The FDA's CRL indicated that the application could not be approved in its current form due to a lack of substantial evidence of effectiveness [2][8] - The FDA noted that ONS-5010 did not meet the primary efficacy endpoint in the NORSE EIGHT trial, necessitating additional confirmatory evidence to support the application [3][8] Next Steps for the Company - The CEO of Outlook Therapeutics expressed disappointment but stated intentions to meet with the FDA for further clarification on approval requirements for ONS-5010 [5] - The company plans to continue expanding into European markets, having already received approval for Lytenava (bevacizumab gamma) for wet AMD in the EU and the U.K. [6][7] Clinical Trial Insights - The FDA acknowledged that the NORSE TWO trial met its safety and efficacy endpoints, but cited several issues including CMC concerns and open observations from pre-approval inspections as reasons for not approving the application [4][8] - The FDA has indicated that a second adequate and well-controlled clinical trial is required, suggesting a non-inferiority study comparing ONS-5010 to ranibizumab [9]

Core Insights - Madrigal Pharmaceuticals (MDGL) received conditional marketing authorization from the European Commission for Rezdiffra (resmetirom) to treat adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced liver fibrosis [1][7] - Rezdiffra is the first and only therapy approved for MASH in the European Union, based on positive data from the pivotal phase III MAESTRO-NASH study [2][3] - The first European launch of Rezdiffra is anticipated in Q4 2025, starting in Germany, and it is already included as a first-line treatment in European MASH guidelines [3] Market Performance - Year-to-date, shares of Madrigal have increased by 23%, outperforming the industry average rise of 9.1% [4] - Despite the EU approval, MDGL stock fell by 3% on August 19 due to investor concerns over competition from Novo Nordisk's Wegovy [6][7] Competitive Landscape - Wegovy, a GLP-1 drug from Novo Nordisk, received accelerated approval from the FDA for treating noncirrhotic MASH, marking it as the first GLP-1 therapy approved for this condition [8][9] - Analysts suggest Wegovy's established safety profile and benefits in metabolic disease may position it as a preferred treatment for MASH, potentially impacting Rezdiffra's market position [9] Sales and Demand - Rezdiffra's commercial launch in the U.S. has shown strong initial demand, generating sales of $212.8 million in Q2 2025, a sequential increase of nearly 55% [11] - As of June 30, 2025, over 23,000 patients are currently receiving treatment with Rezdiffra [11]

Core Insights - The U.S. FDA has approved Insmed Incorporated's Brinsupri (brensocatib) as the first and only treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children aged 12 and older [1][5] - Approximately 500,000 individuals in the U.S. are diagnosed with NCFB, highlighting a significant patient population for this treatment [2] - Clinical trials demonstrated that Brinsupri significantly reduced the annual rate of exacerbations by 21.1% and 19.4% for the 10 mg and 25 mg doses, respectively, compared to placebo [3] - The treatment also met secondary endpoints, including prolonging the time to first exacerbation and increasing the proportion of patients remaining exacerbation-free [4] - The safety profile of Brinsupri was generally consistent across studies, with some increased incidence of gingival and periodontal adverse reactions noted in the WILLOW study [5] - Insmed plans to file for approval in Japan in 2025, with commercial launches anticipated in 2026 pending regulatory approvals [5] - Following the FDA approval, Insmed's stock price increased by 6.20% to $119.89 [6]

Core Insights - Verastem, Inc. (VSTM) is viewed positively despite challenges related to data readouts and drug approval uncertainty [1] Company Analysis - The company operates in the biotech sector, focusing on drug development and clinical trials [1] - There is a commitment to educating investors about the scientific aspects of the business, emphasizing the importance of due diligence in biotech investments [1]

Core Insights - Incyte's supplemental new drug application (sNDA) for ruxolitinib cream has had its FDA review period extended by three months to September 19, 2025, to allow for additional data review [1][2][7] - The sNDA aims to secure approval for treating children aged 2-11 years with mild to moderate atopic dermatitis (AD) [1][4] - Ruxolitinib cream, marketed as Opzelura, is already approved for patients aged 12 and older for the treatment of non-segmental vitiligo [3][5] Company Performance - Year-to-date, Incyte's shares have decreased by 0.9%, while the industry has seen a decline of 2.5% [2] - First-quarter net revenues from Opzelura cream reached $119 million, reflecting a 38% increase [5] Clinical Data - The sNDA is supported by data from the phase III TRuE-AD3 study, which demonstrated that a higher percentage of patients treated with Opzelura achieved treatment success compared to those using a non-medicated cream [4][7] - The study also met a secondary endpoint, with at least 75% improvement in the Eczema Area and Severity Index (EASI75) at Week 8 [4] Portfolio Diversification - Incyte is actively working to diversify its portfolio and reduce reliance on its leading drug, Jakafi (ruxolitinib) [8] - The company has seen strong sales across all indications, with recent approvals of drugs like Pemazyre, Monjuvi, and Tabrecta contributing to this diversification [10] Competitive Landscape - Jakafi faces increasing competition, particularly from GSK's Ojjaara, which reported strong sales growth of £112 million in the first quarter of 2025 [13] - The patent protection for Jakafi is expected to expire in the coming years, which may impact future revenue [14]

Core Insights - GSK's new drug application for linerixibat has been accepted by the FDA for review, with a decision expected on March 24, 2026 [1][7] - If approved, linerixibat could fulfill a significant unmet medical need for patients suffering from cholestatic pruritus associated with primary biliary cholangitis (PBC) [2][5] GSK's Drug Development - The NDA for linerixibat is based on positive results from the phase III GLISTEN study, which demonstrated significant improvement in cholestatic pruritus and related sleep interference compared to placebo [4][7] - Cholestatic pruritus is a common symptom of PBC, a rare autoimmune disease that can lead to liver failure [5] Competitive Landscape - Other companies are also pursuing treatments for PBC, including Gilead Sciences, which received accelerated approval for seladelpar in August 2024 [8] - Seladelpar was acquired by Gilead for $4.3 billion and is now part of their liver disease portfolio [9] - Mirum Pharmaceuticals is developing volixibat, another oral IBAT inhibitor, currently in phase IIb trials [9]