Roche’s sefaxersen hits its Phase III IgAN endpoint with significant proteinuria reduction. See what the result means for regulators and competitors.
FDA approval has transformed zilganersen from an experimental antisense programme into the first disease-modifying medicine for Alexander disease. For Ionis Pharmaceuticals, however, ZANVASTRO is also a test of whether years of RNA-targeted neurology research can translate into a sustainable independently commercialized franchise.
Zanvastro finally gives Alexander disease a targeted therapy. Its next challenge is finding patients and funding a $1.14 million annual regimen.
Novartis and Ionis say pelacarsen substantially reduced lipoprotein(a) but failed to reduce major cardiovascular events in the 8,323-patient Phase 3 Lp(a)HORIZON trial.
The FDA has approved Ionis Pharmaceuticals’ ZANVASTRO for pediatric and adult Alexander disease after a 54-patient study showed significant stabilization of gait at 61 weeks.
See why the CARDIO-TTRansform miss complicates eplontersen’s ATTR-CM path and tests gene silencing on top of stabilizer therapy.
Roche Holding AG has discontinued development of tominersen and RG6496, removing two investigational huntingtin-lowering medicines from its neurology pipeline after separate setbacks emerged almost simultaneously. The Phase 2 GENERATION HD2 trial showed that tominersen lowered important biological markers but did not meaningfully slow clinical decline, while the Phase 1 POINT-HD study of RG6496 was halted
Discover why Ionis' REVEAL enrollment turns obudanersen into a decisive Phase 3 test for Angelman syndrome treatment.
Tryngolza is no longer confined to the rare familial chylomicronemia syndrome market. Its broader United States approval gives clinicians the first therapy specifically shown to reduce acute pancreatitis risk in adults with severe hypertriglyceridemia, although questions surrounding dose selection, hepatic monitoring, reimbursement and long-term use will influence how quickly the treatment changes practice.
Find out how Praxis’ FDA Breakthrough Therapy Designation for elsunersen could reshape SCN2A-DEE epilepsy treatment and ASO development.