Orladeyo now available to treat HAE patients in Ireland
Therapy is first once-a-day, oral HAE prophylactic, Biocryst says
Orladeyo (berotralstat), an oral therapy designed to reduce or prevent hereditary angioedema (HAE) attacks, is now available in Ireland.
The Irish Health Services Executive (HSE) recommended Orladeyo as a prophylactic, or preventive, treatment for HAE in adults and adolescents, ages 12 and older. Biocryst Pharmaceuticals, the company marketing the therapy, said it is the first oral, once daily, prophylactic treatment of its kind.
“The positive HSE recommendation of ORLADEYO broadens access to modern prophylaxis, providing greater choice for prescribing physicians and potentially a better quality of life for HAE patients in Ireland,” Charlie Gayer, chief commercial officer of Biocryst, said in a company press release.
HAE is a genetic disorder characterized by recurrent swelling attacks driven by the excessive production of a signaling molecule called bradykinin. Orladeyo is designed to lower bradykinin levels by inhibiting an enzyme that’s responsible for its production.
Orladeyo was approved in the European Union in 2021 as a preventive treatment to decrease the frequency of attacks in HAE patients, ages 12 and older. It has also been approved in the U.S. and other countries for the same indication.
Clinical trial data show fewer attacks
The approvals were largely based on data from the Phase 3 APeX-2 clinical trial (NCT03485911), which evaluated Orladeyo’s safety and efficacy against a placebo in 121 HAE patients. Data from the trial’s initial phase, which compared the effects of Orladeyo to a placebo over approximately six months, patients taking Orladeyo were found to experience significantly fewer swelling attacks.
Long-term follow-up data from the APeX-2 trial indicated that patients continued to experience low attack rates for up to two years. Additional analyses from APeX-2 and other studies also showed that patients who transitioned from injectable medications to Orladeyo generally expressed higher satisfaction with the oral treatment.
Real-world data from the U.S. presented earlier this year supported these findings, showing that the treatment effectively maintains low swelling attack rates over time, regardless of disease severity or previous preventive treatments.
Biocryst is conducting a Phase 3 clinical trial called APeX-P (NCT05453968) to evaluate the safety and pharmacological properties of Orladeyo in children ages 2-11 with HAE. The study aims to determine the appropriate dose of the therapy for pediatric patients, while assessing the treatment’s safety profile in this younger patient population.
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