Breaking News
- The FDA has published 89 complete response letters (CRLs) in real time for the first time, marking a new era of transparency: https://www.fda.gov/news-events/fda-newsroom/press-announcements
- The agency also issued new Rare Disease Evidence Principles: https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles
- Approvals landed in hematology: Wayrilz (rilzabrutinib) for chronic/persistent immune thrombocytopenia: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia
- Vonvendi (recombinant VWF) expanded to children and adult prophylaxis: https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children
- In Europe, the EMA’s PRAC launched a safety review of levamisole: https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-1-4-september-2025
Industry Trends
- ClinicalTrials.gov’s PRS system is undergoing modernization: https://www.nlm.nih.gov/pubs/techbull/so25/so25_ct_prs.html
- Release notes: https://clinicaltrials.gov/about-site/release-notes
- Modernization hub: https://clinicaltrials.gov/about-site/modernization
- NIH announced flexibilities for BESH registration/results reporting: https://grants.nih.gov/news-events/nih-extramural-nexus-news/2025/07/flexibilities-for-registration-and-results-reporting-of-prospective-basic-experimental-studies-with-human-participants
- NIH Weekly Index: https://grants.nih.gov/grants/guide/weeklyindex.cfm
- University of Rochester posted a new policy (Sept 2025): https://www.urmc.rochester.edu/MediaLibraries/URMCMedia/Policy-Public-Disclosure-Clinical-Trials-Sept-25-2025.pdf
Approvals
- Wayrilz approval: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia
- Vonvendi expanded approval: https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children
Regulatory Updates
- FDA’s “What’s New” in Drugs page: https://www.fda.gov/drugs/news-events-human-drugs/whats-new-related-drugs
- FDA “Green List” initiative for illegal GLP-1 ingredients: https://www.fda.gov/news-events/fda-newsroom/press-announcements
- EMA levamisole review: https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-1-4-september-2025
M&A and Licensing
- Novartis signed an up-to-$5.2B deal with Argo Biopharma: https://www.reuters.com/business/healthcare-pharmaceuticals/novartis-signs-up-52-billion-licensing-deal-with-argo-biopharmaceutical-heart-2025-09-03/
- Financial Times coverage: https://www.ft.com/content/d57325e4-5bc1-447c-a981-80820e17628a
- Sanofi’s $470M acquisition of Vigil Neuroscience: https://www.reuters.com/markets/deals/sanofi-acquire-vigil-neuroscience-470-million-deal-2025-05-21/
- Deal flow context ($100B+ in 2025): https://www.reuters.com/business/healthcare-pharmaceuticals/major-deals-involving-us-drugmakers-biotechs-over-past-decade-2025-07-09/
Other R&D News
- Akeso reported survival benefit in lung cancer trial: https://www.statnews.com/2025/08/26/akeso-says-its-closely-watched-drug-improved-patient-survival-in-a-lung-cancer-trial/
- Summit Therapeutics’ ivonescimab preview: https://www.statnews.com/2025/05/15/summit-therapeutics-ivonescimab-lung-cancer-preview/
- Zymeworks dropped a T-cell engager program: https://www.fiercebiotech.com/biotech/zymeworks-drops-clinical-stage-t-cell-engager-after-assessing-benefit-risk-profile
Tech Breakthroughs
- AI adoption in clinical trial operations/design: https://www.appliedclinicaltrialsonline.com/view/clinical-trial-innovations-gain-adoption
- AI & RWE master protocols: https://www.appliedclinicaltrialsonline.com/view/ai-ml-real-world-evidence-master-protocols-trial-success
- Oxford Vaccine Group awarded £118M for AI-powered human challenge trials: https://www.ft.com/content/e21af386-2171-4d2d-a4a6-9d52761261ff
Future Projections
- China-origin licensing deals expected to accelerate (Novartis–Argo as template): https://www.reuters.com/business/healthcare-pharmaceuticals/novartis-signs-up-52-billion-licensing-deal-with-argo-biopharmaceutical-heart-2025-09-03/
- Rare Disease Evidence Principles to shape new filings: https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles
- ClinicalTrials.gov PRS enhancements increasing scrutiny: https://www.nlm.nih.gov/pubs/techbull/so25/so25_ct_prs.html
| Category | Key Highlights | Links |
|---|---|---|
| Breaking News | FDA CRLs, Rare Disease Principles, Wayrilz, Vonvendi, EMA levamisole review | FDA press | Rare Disease Principles | Wayrilz | Vonvendi | EMA review |
| Industry Trends | PRS modernization, NIH flexibilities, URMC policy | PRS Hub | PRS Bulletin | NIH Flexibility | URMC Policy |
| Approvals | Wayrilz (ITP), Vonvendi expanded use | Wayrilz | Vonvendi |
| Regulatory Updates | FDA “What’s New,” Green List, EMA safety review | FDA What's New | Green List | EMA review |
| M&A / Licensing | Novartis–Argo $5.2B, Sanofi–Vigil $470M, $100B+ YTD deals | Novartis–Argo | FT Coverage | Sanofi–Vigil | Deal Trends |
| R&D News | Akeso lung cancer, ivonescimab, Zymeworks exit | Akeso | Ivonescimab | Zymeworks |
| Tech Breakthrus | AI adoption, Oxford Vaccine AI trial | AI Adoption | AI & RWE | Oxford Vaccine |
| Future Outlook | China licensing, rare-disease filings, PRS scrutiny | China Licensing | Rare Disease | PRS Bulletin |
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