Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema скачать в хорошем качестве

Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema

Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE). HAE is a condition where people have recurrent episodes of severe swelling of the skin and mucous membranes. These attacks generally become more frequent after puberty, and continue throughout life, often affecting the skin, gastrointestinal tract, and upper airway. While skin swelling can cause pain, dysfunction, and disfigurement, it is not considered dangerous. When the gastrointestinal tract is involved, this may cause symptoms such as nausea, vomiting, diarrhea, and abdominal pain. The upper airway is less commonly affected, but can cause upper airway obstruction. Attacks may involve one area or a combination of areas of the body and typically go away on their own within 2 to 5 days. While people with HAE have reported various triggers of attacks, common triggers for attacks include emotional stress, physical stress, and dental procedures. HAE may be caused by genetic changes in the SERPING1 gene or in the F12 gene. Donidalorsen is an RNA-targeted therapy designed to target plasma prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with HAE. Recently, new long-term data was presented at the American College of Allergy, Asthma, & Immunology (ACAAI) Annual Scientific Meeting 2025. This data builds on the clinical evidence that supported the approval of donidalorsen for the treatment of patients ages 12 years and older with HAE in August 2025. The OASISplus OLE cohort enrolled adult and adolescent patients continuing from the Phase 3 OASIS-HAE pivotal trial, who received donidalorsen every four (Q4W) or every eight weeks (Q8W). At Week 52 in the OLE, DAWNZERA demonstrated a 94% and 95% mean attack reduction from baseline for patients in the Q4W and Q8W dosing groups, respectively. Additionally, 97% patients reported well-controlled disease as measured by the Angioedema Control Test at week 52 compared to baseline. OASISplus also included a switch cohort evaluating donidalorsen Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat. At week 52, patients who switched to donidalorsen experienced a 68% improvement in monthly HAE attack rate compared to baseline with prior prophylactic therapy, with 90% reporting well-controlled disease as measured by the Angioedema Control Test. In the Phase 2 OLE study, patients treated with donidalorsen Q4W showed a 97% mean HAE attack rate reduction over four years. The median attack-free interval was 2.7 years, and 71% of patients were attack-free for more than one year. Additionally, donidalorsen demonstrated a favorable long-term safety and tolerability profile across all studies. The majority of treatment-emergent adverse events were mild or moderate. Chapters: Introduction 00:00 HAE Overview 1:38 Dawnzera Overview 3:40 Long-Term Data 6:13 Take Home Message 13:42