Japan Approved Orladeyo for Under-Twelves in an Open-Label Interim Analysis of 29 Children
The molecule has been approved in Japan since 2021. What is new is a formulation a two-year-old can swallow, and that is the commercial move.
Rare Diseases
Rett Syndrome Gets Its First EU Treatment. Thirty Pricing Negotiations Start Now.
First and only is not the same as available. One authorisation covers 30 jurisdictions, each of which now prices the drug separately.
The Number Everyone Will Quote About European Launches Rests on Eleven Products
The 22.9% figure driving coverage of European launch delays is 48 products falling to 37. The pricing finding underneath it is stronger and barely quoted.
Two First-Ever Rare Disease Approvals Landed the Same Day. Both Were Measured by Proxy.
Pasatru’s flare benefit is clinician-assessed only. GENGLYCOS is indicated to reduce cornstarch intake. Two firsts, both measured a step from the patient.
Novartis Declines Canadian Reimbursement for Vanrafia Five Weeks After Health Canada Approved It
Health Canada cleared Vanrafia on a Week 36 proteinuria readout. Novartis will not take that same package into Canadian pricing review.
Jazz Paid $820 Million Upfront for a Phase 1b/2a Because the FDA Pathway Came With It
QuickDeal read/Rare Diseases/Small Molecule Jazz Paid $820 Million Upfront for a Phase 1b/2a Because the FDA Pathway Came With It Athithi Verma· 11 August 2026· 3 min read· Synopulse Jazz Pharmaceuticals will acquire privately held Actio Biosciences for $820 million upfront plus up to $500 million in approval and sales milestones, a maximum of $1.32 … Read more
Scholar Rock Filed With Two Fill-Finish Sites. One Just Failed, and the Date Held.
Catalent Indiana failed its FDA inspection. Scholar Rock had named a second fill-finish site in the same BLA, so the PDUFA date did not move.
Congress Exempted the Medically Frail. The Coding System Cannot Find Them.
Fewer than 500 of 10,000 rare diseases have an ICD-10 code. The Medicaid frailty exemption is verified against codes most patients never had.
An FDA Panel Voted 9-3 Against Capricor on Cardiomyopathy and Never Voted on the Endpoint That Passed
The ballot was the outcome. A drug’s strongest dataset sitting off the voting question is not a footnote, it is the whole result.
Italy Ran an Innovation Test for Eight Years.
Italy graded new medicines full, conditional or not innovative for eight years. The 2025 reform abolished the middle grade, because almost nothing ever graduated.