Rare Disease Catalyst Report: Non-Oncology Pipeline & Milestones: October 2026
Shionogi bought into Niemann-Pick type C for $2bn. Beren’s rival faces the FDA on 17 November, and Rafael’s has just filed.
NeuroPulse
DealPulse Signal
The Beats
Analysis
The Briefs
The FDA Will Let One Licence Cover Patient-Specific Gene Edits, and CMS Is Taking Its Medicaid Gene Therapy Deals Beyond Sickle Cell
NICE Backed Arrowhead’s Plozasiran Eight Days After UK Authorisation, as a Cost Match for 57 to 114 People in England
Canada’s Drug Agency Has Drafted a Yes on Spinraza for Adults With SMA, Four Years After Telling Provinces Not to Pay
Enhertu Won EU Approval 39 Days After Its CHMP Opinion, and Payers Now Hold a Label Wider Than the Trial
China Approved Bayer’s Asundexian, the First Factor XIa Inhibitor, on a 26% Stroke Reduction, Three Years After It Lost to Apixaban
