Donidalorsen for Long-Term Prophylaxis of Hereditary Angioedema Attacks: Results from the OASISplus Open-Label Extension Cohort at Year 1 - PubMed
8 hours ago
- #donidalorsen
- #prophylaxis
- #hereditary angioedema
- Donidalorsen is an antisense oligonucleotide used for long-term prophylaxis of hereditary angioedema (HAE) attacks in patients aged 12 years and older.
- The OASISplus open-label extension study evaluated the efficacy and safety of donidalorsen over one year, following the phase 3 OASIS-HAE trial.
- Patients received donidalorsen either every 4 weeks (Q4W) or every 8 weeks (Q8W), with some switching from Q8W to Q4W if not attack-free.
- Results showed a 94% (Q4W) and 95% (Q8W) reduction in mean HAE attack rates from baseline, along with clinically meaningful improvements in quality of life (AE-QoL).
- The safety profile was acceptable, with 27% of patients reporting treatment-related adverse events, mostly injection-site reactions, none of which were serious.
- The study concluded that donidalorsen provides sustained reductions in HAE attack rates and improves quality of life with an acceptable safety profile over one year.