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.
Rare disease
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.
OrphanPulse Wk 29: Ionis Takes SMA-Proven Antisense Into Dravet Syndrome
A week defined by first-in-human starts across rare genetic disease, led by antisense entering Dravet syndrome from the field that already rewrote spinal muscular atrophy. Around it, first patients dosed in rare vascular and cardiomyopathy programs with near-empty competitive fields, a first-in-class readout in heparin-induced thrombocytopenia, and orphan designations that quietly reshape the economics behind them.
Reimbursement Series: CMS and Europe Reimbursement Pathway Accelerates Innovation
In a transformative week for market access, the CMS and Europe reimbursement pathway expansion delivered several pivotal coverage and pricing decisions reshaping patient access across cardiovascular, hepatic, and oncology therapies. From CMS’s national determinations on breakthrough devices to European HTA approvals in rare disease and cancer, these developments underscore a growing transatlantic alignment between regulators … Read more
Alexion Strikes Bold AI Partnership for Rare Disease Detection
AI partnership targets rare bone disease affecting 1 in 6,000 patients.