Rare diseases
Oak Hill Bio is developing the antisense therapy rugonersen, which it licensed from Roche in 2025, for Angelman syndrome.
In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor’s assertion that a Phase 3 trial met its primary outcome measures.
After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
Facing pressure around the world to withdraw the rare kidney disease drug Tavneos, Amgen has launched a robust defense with real-world evidence to support the product’s benefit-risk profile.
With Phase 3 data showing what analysts called a “potentially best-in-class profile,” Arrowhead Pharmaceuticals plans to submit for FDA approval of plozasiran by the end of the year—hoping to follow to market Ionis Pharmaceuticals’ Tryngolza, which got the regulator’s nod in severe hypertriglyceridemia last month.
A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations.
Agios Pharmaceuticals’ sickle cell disease candidate didn’t prove any better than Novo Nordisk’s investigational PK activator or the biopharma’s own asset that is currently under FDA review for the indication.
Spero Therapeutics is putting over $1 billion on the line in exchange for rights to develop SP001, an anti-CD40L antibody, for IgG4-related disease. A Phase 2 study is planned for the second quarter of 2027.
The trials, which were testing the tyrosine kinase inhibitor masitinib, had previously been paused. AB Science’s decision not to resume them was a matter of prioritization, not safety, the biotech said.
Regulators on both sides of the Atlantic are pushing for the withdrawal of the rare disease treatment that accounted for just 1% of Amgen’s 2025 revenue. Nevertheless, Amgen continues to defend the medicine, which was acquired in the $3.7 billion buyout of ChemoCentryx.
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