A new study revealed high levels of satisfaction among patients with hereditary angioedema (HAE) who switched from injectable prophylactic treatments to berotralstat (Orladeyo®) monotherapy. The results of the study are being presented as a poster at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit.

“Statistically significant improvements were observed in convenience and global satisfaction scores in patients who switched from lanadelumab (Takhzyro®) and SC C1-INH [C1 esterase inhibitor subcutaneous, Haegarda®] to oral berotralstat monotherapy, suggesting a positive experience using an oral HAE prophylactic therapy,” the authors said.

From baseline to month 12, convenience score improved by 33.4 points, while global satisfaction score improved by 20.8 points in patients who switched to berotralstat monotherapy. The largest improvement was observed in the convenience domain, which improved by 26.4 points in patients who switched from lanadelumab to berotralstat monotherapy and by 42.2 points in patients who switched from SC C1-INH to berotralstat monotherapy.


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Moreover, berotralstat was as effective as patients’ prior prophylactic therapy, as shown by the analysis of effectiveness scores, which remained consistently high from baseline to month 12.

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The study, conducted by Bernstein et al, enrolled 34 patients who discontinued their prior injectable long-term prophylactic treatment and switched to berotralstat 150 mg monotherapy. Most (68%) participants discontinued their prior treatment before study enrollment, while 32% of participants discontinued their prior treatment after initiating berotralstat monotherapy.

Berotralstat, also known as BCX7353, was approved in December 2020 for prophylaxis of HAE attacks in patients at least 2 years old.

Reference

Bernstein JA, Desai B, Tomita DK, et al. Improved patient satisfaction with berotralstat in patients switching from injectable hereditary angioedema (HAE) prophylactic treatments. Poster presented at: National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit; October 17-18, 2022; Washington, DC.