Approvals· August 22, 2026 · 19 min read
Zilganersen's September 22 PDUFA: Alexander Disease ASO & Intrathecal Access
By Ran Chen#FDA Approval#NDA Approval#Priority Review
Topic
Rare Disease
Rare-disease commercialization, orphan status, access barriers, evidence uncertainty, and launch watchlists.
Approvals· August 21, 2026 · 19 min read
Pasatru's FOP Approval: What Garetosmab's $1.4M Launch Means vs Sohonos
By Ran Chen#FDA Approval#BLA Approval#Rare Disease
Pricing & Access· August 18, 2026 · 16 min read
DTX401's August 23 PDUFA: Payers Face a Cornstarch-Replacement Gene Therapy
By Ran Chen#Gene Therapy#Rare Disease#Medicaid
Pricing & Access· August 17, 2026 · 25 min read
Tavneos on Formularies as FDA Moves to Withdraw: The Coverage Dilemma
Specialty· August 14, 2026 · 20 min read
Spinal Muscular Atrophy Treatment Access Landscape: Spinraza, Itvisma, & Evrysdi
By Ran Chen#Rare Disease#Gene Therapy#Antisense
Pricing & Access· August 13, 2026 · 20 min read
Epilepsy Treatment Access (2026): From $0.02 Generics to Zorevunersen
By Ran Chen#Antiepileptic Drugs#Antisense#Rare Disease
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· August 2, 2026 · 25 min read
Angelini Pharma's $4.1B Catalyst Acquisition: Rare Disease Deal Anatomy
By Ran Chen#M&A#Rare Disease#Orange Book
Deals· July 30, 2026 · 21 min read
Biogen's $5.6B Apellis Acquisition: Syfovre & Empaveli Q2 2026 Financial Deep Dive
By Ran Chen#M&A#Commercial Strategy#Ophthalmology
Specialty· July 29, 2026 · 24 min read