OrphanPulse Wk 42: Europe Backed Ascendis’ Weekly Achondroplasia Shot Ahead of the First Pill
Europe now has a daily achondroplasia injection on the market, a weekly one recommended, and an oral drug planning to file.
OrphanPulse
Weekly rare-disease beat.
OrphanPulse Wk 41: BridgeBio Won Priority Review for the First Oral Achondroplasia Drug
Infigratinib would be the first oral achondroplasia drug, against a daily injection and a weekly one already on the market.
OrphanPulse Wk 40: A First Approval in MCT8 Deficiency, and a Missed Pivotal Headed for Filing
One first approval, and one missed pivotal headed for filing anyway: the week tested what rare disease evidence can carry.
OrphanPulse Wk 39: A 900-Patient Disease Now Has Two Approved Drugs
Mirum licensed Atebrioz five months before its PDUFA date. The approval arrived a day early on a single Phase 2 cohort.
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.
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.
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.