Orladeyo effective in HAE patients of different ages, data show
Preventive treatment found to reduce swelling attacks in children, adults
The use of Orladeyo (berotralstat) — widely approved as a preventive treatment for hereditary angioedema (HAE) — can help reduce the rates of swelling attacks in people with HAE of different ages, according to new data from clinical trials and real-world analyses.
Biocryst Pharmaceuticals, the developer of the oral prophylactic therapy, noted in a company press release that these data “[support] berotralstat use at all stages of life,” with “broad safety and efficacy outcomes observed across all age groups of patients taking Orlaydeyo to prevent HAE attacks.”
The company showcased these findings in a series of oral presentations at the 14th C1-Inhibitor Deficiency & Angioedema Workshop, held last week in Budapest, Hungary.
“These data underscore the potential of our oral, once-daily prophylactic treatment to deliver consistent, meaningful benefits for people living with HAE across all stages of life. From young children to adolescents and adults, Orladeyo continues to show strong efficacy and tolerability, helping patients experience more days free from attacks,” said Helen Thackray, MD, chief research and development officer at Biocryst.
A genetic disorder, HAE is marked by recurrent swelling attacks that are driven by the overproduction of a signaling molecule called bradykinin. Orladeyo works to block the activity of kallikrein, an enzyme that’s involved in bradykinin production.
The therapy is approved in the U.S. and several other countries, as well as in the European Union, to reduce the risk of swelling attacks in people with HAE, ages 12 and older.
Phase 3 trial now testing Orladeyo in children with HAE, ages 2-11
Biocryst is now assessing the efficacy of Orladeyo in younger children with HAE.
An ongoing, open-label, Phase 3 clinical trial called APeX-P (NCT05453968) is testing the therapy in pediatric patients, ages 2 to 11. All participants are being given daily Orladeyo at doses adjusted based on body weight.
Interim trial data were shared at last week’s workshop in a presentation titled “Reduction in Percentage of Days with Angioedema Symptoms Among Young Children Initiating Berotralstat – Interim Results from ApeX-P.”
The findings showed that the percentage of days with HAE symptoms among patients decreased from a mean of 11% before Orladeyo treatment to 4% after three months on the prophylactic.
This reduction was maintained for as long as about one year, according to Biocryst. The company also noted that at least half of the children have been attack-free each month while on Orladeyo. Before enrolling in APeX-P, they had a mean of 18 missed school days per year.
The most common safety issues reported in APeX-P so far include the common cold, upper respiratory tract infection, and headache, according to Biocryst. The trial is slated to run through early 2027.
These clinical and real-world results give … confidence that we can continue to have a very positive impact on advancing care for all those living with HAE, regardless of their age.
A separate presentation was titled “Tolerability and Effectiveness of Berotralstat for Prophylaxis in Adolescents with Hereditary Angioedema: subgroup analysis of the Berolife Study.” It detailed the outcomes from Orladeyo treatment in a subgroup of adolescents, ages 12 to 17, who received the therapy as part of a real-world study in France called Berolife.
These findings showed that, six months after starting treatment, the median rate of swelling attacks decreased from 2.25 per month to 0.55 per month. This reduction in the rate of monthly attacks was sustained out to 1.5 years on Orladeyo. The therapy’s safety profile in adolescents was consistent with that seen in previous clinical trials and in the overall population of HAE patients taking part in Berolife, per the company
A third presentation, titled “Berotralstat for the Prevention of Hereditary Angioedema Attacks: Results from the Italian Expanded Access Program,” reported outcomes from HAE patients who received Orladeyo as part of an expanded access program in Italy. Over the first six months of treatment, measures of symptom control and life quality gradually improved among the 12 patients with available data, the findings indicated.
Thackray noted that these new data show the benefits of Orladeyo use across many different life stages.
“These clinical and real-world results give me confidence that we can continue to have a very positive impact on advancing care for all those living with HAE, regardless of their age,” Thackray said.
About the Author
