The trials did not show a statistically significant reduction in annualized clinical fracture rates compared to placebo or bisphosphonates
The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026
Despite some promising subgroup signals, the company indicated it will not pursue further psychiatric trials
Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation
The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia
The decision is based on robust clinical evidence from the VOYAGE and EXCURSION studies
The study found that nerandomilast effectively slowed lung function decline in patients, with discontinuation rates similar to placebo
Adverse events were consistent with valbenazine’s established safety profile
The therapy also drove additional weight loss at the higher 1.8 mg dose without plateauing and maintained a favorable tolerability profile
FOP is a rare genetic disorder caused by mutations in the ALK2 kinase
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