Approvals· September 4, 2026 · 36 min read
Lirafugratinib's Sept 27 PDUFA Does Not Rewrite Pemazyre PAs or Lytgobi CDx
By Ran Chen#PDUFA Date#FDA Approval#NDA Approval
Topic
Rare Disease
Rare-disease commercialization, orphan status, access barriers, evidence uncertainty, and launch watchlists.
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
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 29, 2026 · 15 min read
Lisraya's DM Label: Boxed-Warning 30 mg Tablet, Table 3 Split, Unapproved 15 mg
By Ran Chen#FDA Approval#JAK Inhibitor#Dermatology
Approvals· August 29, 2026 · 24 min read
Mimrylo for PV Erythrocytosis: 76.9% Week 20-32 Response and Platelet Rises
By Ran Chen#FDA Approval#Hematology#Coverage Access
Approvals· August 27, 2026 · 20 min read
Imaavy's wAIHA Approval Is a Steroid-Gated, q4w Indication: J9256 Does Not Auto-Cover It
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
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