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.
Small Molecule
A Dual MYC and GSPT1 Degrader Extended Survival by Over 300% in One Preclinical Model
The loop is the discovery, not the degrader. If GSPT1 supports MYC translation, a GSPT1-only degrader should permit MYC to recover.
China Will Pay One Price for 158 Conditions, Whatever the Hospital
Same disease, same payment across tiers. Of 31 DRG categories, 27 are internal medicine, where drug cost rather than theatre time drives the case.
RK-251 Was Tested Against One Cancer Cell Line. The Problem It Was Built to Solve Is Bioavailability.
FindingsScience read/Oncology/Small Molecule RK-251 Was Tested Against One Cancer Cell Line. The Problem It Was Built to Solve Is Bioavailability. Athithi Verma· 25 August 2026· 2 min read· Synopulse What they did A research article in the Journal of Medicinal Chemistry, published 23 July 2026 across pages 17342 to 17365. Kesarwani and colleagues at IIT … Read more
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.
The Effect Size Is 0.81. The Number That Decides the Business Is 6.4 Hours.
Effect size 0.81 settles the efficacy question. The number that decides the business is 6.4 hours of supervised monitoring per dose.
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
Tau does its damage inside mitochondria, independent of tangles and microtubules. The field’s imaging toolkit was built to see neither.
Tau does its damage inside mitochondria, independent of tangles and microtubules. The field’s imaging toolkit was built to see neither.
The FDA Approved Orzeyful and Handed the Approval Date to the DEA
FDA’s letter says the approval date is whenever the DEA publishes scheduling. Takeda holds a first-in-class approval it cannot sell yet.
NICE Backed Acalabrutinib and Venetoclax Without Obinutuzumab, and the Omission Is the Decision
AMPLIFY tested acalabrutinib and venetoclax with and without obinutuzumab. NICE recommended the doublet, which is a decision about infusions.