D2.531 - Expert Panel Discussion on a New Oral On-Demand Treatment in Hereditary Angioedema

Despite Hereditary Angioedema (HAE) guideline recommendations to consider on-demand treatment (OD) for all attacks, most patients delay or avoid using injectable OD due to logistical challenges and injection-related anxiety. Sebetralstat is the first oral OD treatment approved for HAE.

Objectives: 1) to identify patient characteristics and unmet needs prior to initiating sebetralstat 2) to understand the process of injectable OD discontinuation and transitioning patients to sebetralstat; 3) to review patient feedback on sebetralstat and identify similarities and differences between patient and physician perspectives.

Nine expert HAE treating physicians reviewed HAE patient charts prior to a roundtable discussion of the results. Patient feedback was shared by treating experts where available. Qualitative analysis is presented here.

Of 26 patient charts reviewed mean age was 41 yrs, 73% females, 92% Type 1; 92% on LTP; most used OD was Icatibant 63%; most used LTP was lanadelumab 46%. Most attacks were moderate (59%); 18% required ED visit (based on 17 patients who had this information in the chart). 54% of physicians reported patients using their injectable OD ‘all’ or ‘most’ of the time prior to starting sebetralstat (53% according to patients). According to patient feedback, they are more likely to treat more attacks, more promptly with sebetralstat (Figure).

Top 2 factors influencing patients’ decision to start sebetralstat: oral administration preference (65%) and anxiety/discomfort with injectable therapy (41%), according to physicians; oral administration preference (53%), impact of injectable therapy on QoL (24%) and ability to recognize symptoms (24%), according to patients. 87% of physicians were “likely”/“very likely” to prescribe sebetralstat as the only OD for patients with a history of laryngeal attacks; most experts noted that the oral route of administration, ease of use, and demonstrated safety and efficacy make sebetralstat well suited for these patients. In patients satisfied with sebetralstat, with low rate of attacks, the majority of experts would also consider discontinuing LTP.

Patients are likely to treat more attacks and to treat them more promptly with sebetralstat compared to injectable OD. Most experts indicated sebetralstat was well suited to be prescribed as sole therapy for patients with history of laryngeal attacks. The majority would also consider discontinuing LTP in patients with low rate of attacks who are satisfied with sebetralstat.