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.
Rare disease
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.
OrphanPulse Wk 33: The Competitor Got a PDUFA Date Five Days Later
Five days after Tarsus paid $450 million for a Stargardt asset reading out in 2029, the competitor got a PDUFA date of 12 February 2027.
OrphanPulse Wk 32: Tarsus Paid $450M Upfront for a Readout in 2029
Tarsus committed $450 million at signing for a Stargardt asset whose pivotal trial reads out in 2029, while a competitor is already filing.
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.
OrphanPulse Wk 30: CHMP Backs a Drug It Refused Twice
Nezglyal was refused in February 2024 and again in May. This week the same committee recommended it, and children with cerebral ALD get an oral option.
JCR Gets Its Brain-Penetrating Hunter Therapy Out of Japan for the First Time, Through the UAE
IZCARGO, a therapy engineered to cross the blood-brain barrier, won its first marketing authorization outside Japan five years after launching there.
Crysvita Reaches One-Month-Old Infants in the EU, and Buys Two More Years of Orphan Exclusivity
The European Commission approved Crysvita for infants as young as one month with XLH, a decision that also buys Kyowa Kirin two more years of orphan exclusivity.
A Father’s Liver, One Incision: the First Single-Port Robotic Liver Transplant Lands in a Six-Year-Old
A six-year-old with a rare genetic liver disorder became the first patient to receive a liver transplant through a single robotic incision, donated by his father.