The FDA accepted uniQure's BLA for ifezuntirgene inilparvovec with Priority Review and a May 1, 2027 PDUFA date. The accelerated-approval bid rests on an external natural-history control, not a placebo arm.
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Its $1.275 billion upfront commitment to a Phase 1 program puts the spotlight on oral peptide developers, including Entera as it approaches Phase 3 in osteoporosis.
Gilead Sciences is paying $1.275 billion upfront for an oral IL-23 receptor drug with no efficacy data, staking a claim on an all-oral IBD franchise built around its own emvistegrast.
AstraZeneca's fifth Ultomiris indication arrives on a proteinuria surrogate, while the week-106 eGFR readout still decides whether the drug actually saves kidneys.
In depth
With only about 5% of rare conditions carrying an FDA-approved treatment, the agency is trying to turn case-by-case flexibility into written, predictable defaults. Whether that produces certainty or new rigidity is the open question.
Read the storyArriVent's firmonertinib beat chemotherapy on investigator reads in a 398-patient Phase 3, but the blinded central review it chose returned p=0.0654, and first-line EGFR exon 20 lung cancer now belongs to combinations that keep chemotherapy in the regimen.
CSL is paying $355 million upfront for an antibody whose best evidence is a 26-patient interim readout. The deal prices claudin-1 biology, not proven data, across kidney and liver fibrosis.
PhRMA sued CMS on October 7 over the GLOBE most-favored-nation rule for Medicare Part B. The final rule covers as few as four manufacturers and $440 million in projected savings. The suit's real target is the CMMI test authority beneath it.
Conjugating antisense oligonucleotides to transferrin receptor transport vehicles achieves fifty-fold deeper brain penetration, challenging the standard of invasive spinal punctures.
