Praxis Precision’s Tremor Drug Trial Success: Praxis’s ulixacaltamide met endpoints in two Phase 3 trials for essential tremor, marking one of the first major late-stage wins in the indication. MarketWatch
EO2463 Receives Fast Track Designation: Enterome’s OncoMimics-based immunotherapy demonstrated 46% ORR in interim Phase 2 results in follicular lymphoma, earning FDA Fast Track. Targeted Oncology
TECVAYLI + DARZALEX FASPRO Combo Improves Survival: The MajesTEC-3 Phase 3 study showed improved PFS and OS in relapsed/refractory multiple myeloma. PR Newswire
Industry Trends & Policy Updates
FDA Launches National Priority Voucher Program: First vouchers awarded to nine sponsors on Oct 16, 2025, incentivizing R&D in priority health areas. FDA
NIH $2M Grant to EVOQ Therapeutics: To advance NanoDisc-based therapies for celiac disease and autoimmune disorders. BioSpace
AbbVie’s RINVOQ Label Expanded for Earlier IBD Use: FDA now allows use prior to TNF blockers for ulcerative colitis and Crohn’s. AbbVie News
Orphan Drug Designation Surge: Over 850 orphan drugs in development; FDA grants new rare-disease designations in IPF and glioblastoma. GlobeNewswire
Approvals & Label Expansions
Lurbinectedin Combination Approval: Expanded indication for maintenance use with atezolizumab in extensive-stage SCLC. Wikipedia
Cemiplimab Adjuvant Approval: FDA approves for adjuvant use in high-risk cutaneous squamous cell carcinoma post-surgery/radiation. Wikipedia
The FDA has published 89 complete response letters (CRLs) for the first time, signaling a new era of transparency (FDA press release). The agency will now release CRLs in real time.
In parallel, the FDA unveiled the Rare Disease Evidence Principles, a framework that encourages the use of flexible endpoints, external controls, and adaptive designs in small or heterogeneous populations.
On the approvals front, Wayrilz (rilzabrutinib) gained clearance for adults with chronic or persistent immune thrombocytopenia (FDA approval). Vonvendi (recombinant von Willebrand Factor) secured expanded use to include peri-operative and on-demand treatment in children, as well as prophylaxis in adults (FDA announcement).
Across the Atlantic, the EMA’s PRAC opened a safety review of levamisole, underscoring Europe’s vigilance on older generics (EMA release).
Industry Trends
The ClinicalTrials.gov Protocol Registration System (PRS) is undergoing a modernization overhaul with cleaner workflows and enhanced results editing tools (PRS Tech Bulletin; Release Notes). The goal is to achieve better compliance and reduce friction for sponsors.
The NIH has extended flexibility for prospective basic experimental studies with humans (BESH), providing academic researchers with additional time for registration and results reporting (NIH announcement).
Meanwhile, universities are tightening their own disclosure rules. The University of Rochester rolled out a revised public posting policy, ensuring faster updates for secondary outcomes.
Regulatory Updates
Beyond CRL transparency, the FDA launched a “Green List” to block imports of illegal GLP-1 ingredients (FDA press). On the European side, the review of levamisole serves as a reminder of the continent’s proactive pharmacovigilance.
The agency’s “What’s New” pagefeatures new drug snapshots and label updates, which are posted weekly.
M&A and Licensing
The season’s biggest deal: Novartis signed an up-to-$5.2 billion licensing pact with Argo Biopharma, covering siRNA cardiometabolic assets outside China (Reuters, FT).
Meanwhile, Sanofi’s $470 million acquisition of Vigil Neuroscience is moving toward close this quarter (Reuters).
With pharma/biotech M&A already topping $100 billion YTD, analysts expect the momentum to hold through year’s end (Reuters).
R&D News
Chinese biotech Akeso has reported a promising survival benefit in its lung cancer trial, positioning its PD-1/VEGF bispecific as a potential challenger to Keytruda (STAT). This development is one to watch, as it has the potential to reshape the landscape of lung cancer treatment.
Summit’s ivonescimab remains a candidate to watch as Western readouts approach (STAT preview).
Not all news was positive: Zymeworks' decision to terminate its T-cell engager program after a benefit-risk reassessment has added to the industry's growing concerns around TCEs (FierceBiotech).
Tech Breakthroughs
AI continues to infiltrate drug development. Industry surveys indicate that capabilities are ready, but adoption lags by years (Applied Clinical Trials; AI & RWE protocols).
The most ambitious move: Oxford’s Vaccine Group won £118 million in funding from the Ellison Institute to launch AI-powered human challenge trials for antimicrobial-resistant pathogens (FT).
Future Projections
Expect more China-origin licensing deals in RNA and cardiometabolic disease, modeled on the Novartis–Argo playbook. The FDA’s Rare Disease Evidence Principles are likely to accelerate filings from smaller sponsors (FDA guidance).
With PRS modernization and the FDA’s new disclosure stance, sponsors should prepare for closer scrutiny of protocol amendments, endpoints, and outliers (PRS bulletin).
Summary
Category
Key Highlights
Links
Breaking News
FDA CRLs, Rare Disease Principles, Wayrilz, Vonvendi, EMA levamisole review
