Denmark Said No to a DKK 2.5m CAR-T in January and Yes Once Kite Tied the Price to Response
Denmark turned a January no on Tecartus into a yes by tying price to response, and renewed burosumab on a lower price.
Rare Diseases
Shionogi Paid 29 Times Sales for IntraBio, 17 Days After AQNEURSA’s Second FDA Approval
Shionogi paid about 29 times sales for a drug that gained its second indication 17 days before the deal was signed.
CSL Paid $355 Million and Gave Up 45% of the Profits to Win a Claudin-1 Antibody
No development milestones and no royalty: CSL funds every trial through Phase 3 and gives Alentis 45% of the profits.
NICE Backs Tryngolza for FCS, Giving Sobi a Monthly Successor to Its Own Waylivra
Sobi is moving its FCS franchise to a less frequent, better-tolerated drug, on broader NICE terms than the one it replaces.
FOP Now Has Three Approved Drugs for About 300 US Patients, Two of Them in 37 Days
Three drugs now divide an ultra-rare disease by mechanism, route and age, and the newest competes on convenience and safety.
Europe Approved Leriglitazone for Boys With cALD Two Years After the EMA Recommended Refusal
A drug the EMA refused two years ago is now approved, on a narrow label and under exceptional circumstances, with Europe first.
China Ordered Rare Disease Clinics in Every Province by December and Left Payment Out
Every instruction raises the number of diagnosed patients. None addresses what they can be prescribed, or who pays.
OrphanPulse Wk 38: Two Untreatable Childhood Diseases Got First Therapies in 24 Hours
Seventeen treated children carried a gene therapy to standard full approval. Effect size did the work design could not.
Rare Disease Produced Two First-Ever Approvals in Two Days, and Both of Them Are Neurology
The FDA approved first-ever treatments for Sanfilippo syndrome type A and ataxia-telangiectasia a day apart. Both diseases destroy the nervous system.
Korea’s Reimbursement Delay Is a Staffing Problem, and the Ministry Said So
An industry association found the public agrees with industry. The news came later, from a division director naming headcount as the bottleneck.