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
Weekly rare-disease beat.
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.
OrphanPulse Wk 31: The Panel Voted on a Process, Not a Drug
FDA advisers voted 9 to 3 against Capricor’s deramiocel, three weeks before PDUFA. The argument was not about biology. It was about which SAP counted.
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.
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.