Approvals· September 2, 2026 · 33 min read
Aqneursa's Sept 19 A-T PDUFA Does Not Rewrite NPC-Only PAs or the 15 kg Floor
By Ran Chen#PDUFA Date#FDA Approval#NDA Approval
Topic
Orphan Drug
Orphan drug designation, exclusivity, rare-disease launch planning, payer evidence needs, and access implications.
Approvals· September 1, 2026 · 29 min read
Camzyos's Sept 30 Adolescent PDUFA Does Not Rewrite Adult REMS or Age-18 PA Gates
By Ran Chen#PDUFA Date#FDA Approval#NDA Approval
Approvals· August 31, 2026 · 18 min read
Stelara's Pediatric UC License Does Not Write Wezlana Pediatric IBD Substitution
By Ran Chen#Biosimilars#Interchangeability#Purple Book
Approvals· August 27, 2026 · 20 min read
Imaavy's wAIHA Approval Is a Steroid-Gated, q4w Indication: J9256 Does Not Auto-Cover It
Pipeline· August 19, 2026 · 20 min read
Avexitide LUCIDITY Phase 3: What the First GLP-1 Antagonist Means for PBH Access
By Ran Chen#Pipeline#GLP-1#Clinical Evidence
Deals· August 3, 2026 · 15 min read
Neurocrine's $2.9 Billion Soleno Acquisition: Vykat XR and the Prader-Willi Franchise
By Ran Chen#M&A#Rare Disease#Orange Book
Deals· July 22, 2026 · 18 min read
Vertex's $10B Crinetics Acquisition: Paltusotine and Rare Endocrine Bet
By Ran Chen#M&A#Orphan Drug#Rare Disease
Specialty· July 7, 2026 · 21 min read
Voranigo (vorasidenib) Glioma Access: Pricing, Biomarker Testing, and Payer PA
By Ran Chen#Oncology#Orphan Drug#Prior Authorization
Policy· June 30, 2026 · 18 min read
China Drug Trial Data Protection (NMPA 2026 No.47): Exclusivity and BD Strategy
By Ran Chen#China#NMPA#Regulatory
Biosimilars· June 21, 2026 · 6 min read