Orladeyo approved for reimbursement in the Netherlands
Oral therapy can be used to prevent swelling attacks in HAE patients
Orladeyo is now approved for reimbursement in the Netherlands following a recommendation from healthcare government agency Zorginstituut Nederland.
The treatment is used for routine prevention, or prophylaxis, of swelling attacks in people 12 years and older with hereditary angioedema (HAE).
With this decision, Orladeyo (berotralstat) is available under national reimbursement across all major European countries, “providing HAE patients with access to the first oral, once-daily preventive therapy,” according to Biocryst Pharmaceuticals, the company marketing the therapy.
“From day one, our mission has been to bring [Orlaldeyo] to as many people living with hereditary angioedema as possible. Securing reimbursement in the Netherlands is a proud moment for our team as it means that patients and physicians across all major European countries now have access to a modern, oral prophylactic option to help prevent HAE attacks,” Abid Karim, general manager of Europe at Biocryst, said in a company press release. “This is a great day for the HAE community and for the future of treatment.”
Biocryst seeking regulatory approval for Orladeyo in younger patients
Orladeyo is a daily oral therapy that works by suppressing the enzyme kallikrein, which is involved in the production of bradykinin. This molecule is excessively produced in HAE patients and thought to trigger the swelling attacks that characterize the disease.
This reimbursement decision in the Netherlands follows Orladeyo’s approval in the EU as a preventive treatment to reduce the frequency of swelling attacks in HAE patients 12 years and older. The therapy has received similar approvals in the U.S. and several other countries.
These approvals were primarily supported by results from the Phase 3 APeX-2 clinical trial (NCT03485911), which demonstrated a significant and sustained reduction in the frequency of HAE swelling attacks with continuous Orladeyo treatment. These results have been recently confirmed by real-world data.
While Orladeyo is currently approved for patients 12 years and older, interim data from the ongoing Phase 3 APeX-P clinical trial (NCT05453968) in 29 children, ages 2 to 11, also showed promising results. Specifically, the therapy has been well tolerated and effective in reducing monthly attack rates in pediatric patients, with the mean monthly attack rate dropping from 1.5 on standard care to 0.5 after one month of treatment with Orladeyo, and to 0.3 after one year.
These promising findings supported Biocryst’s efforts to seek regulatory approval for Orladeyo in younger patients. If approved, Orladeyo would become the first oral prophylactic therapy for HAE in children younger than 12.
More recent data in children also showed the proportion of days with HAE symptoms decreased from 11% before treatment to 4% after three months, with about half of the children remaining attack-free each month.
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