Chile OKs Orladeyo to prevent HAE attacks in patients 12 and older
Approval is the preventive therapy’s first marketing authorization in Latin America
Authorities in Chile have approved Orladeyo (berotralstat) to prevent swelling attacks in hereditary angioedema (HAE) patients 12 and older, marking the therapy’s first marketing authorization in a Latin American country.
The preventive, or prophylactic, oral therapy from BioCryst Pharmaceuticals holds similar approvals in the U.S., Europe, Japan, and several other countries worldwide.
“Today’s announcement marks the first approval of Orladeyo in Latin America, which is an important step forward in addressing the significant unmet needs of hereditary angioedema patients who live in the region,” Charlie Gayer, chief commercial officer of BioCryst, said in a press release.
“With this approval, we continue our mission to bring Orladeyo to patients around the world who could benefit from an oral, once-daily prophylactic treatment option to improve control of their HAE attacks,” Gayer said.
In HAE, the overproduction of an inflammatory molecule called bradykinin is responsible for the disease’s characteristic swelling attacks.
Orladeyo is a daily oral therapy that works to suppress the activity of kallikrein, a bradykinin precursor. It’s expected to ease the burden associated with standard injectable treatments.
Its first approvals were supported largely by data from the placebo-controlled Phase 3 APeX-2 trial (NCT03485911) and the open-label Phase 2/3 APeX-S study (NCT03472040).
Findings from these trials indicated that about a year of treatment with Orladeyo reduced the number of monthly HAE attacks, leading to a decreased need for on-demand therapies and improvements in life quality for patients 12 and older.
Orladeyo also was found to be generally safe and well tolerated, with the most common side effects reported being related to gastrointestinal distress.
Real-world and clinical trial data have continued to support Orladeyo’s safety and sustained effectiveness since its first approvals.
Currently, Orladeyo’s approvals cover patients 12 and older. A Phase 3 trial called APeX-P (NCT05453968) is underway to assess the therapy’s effects in children with HAE, ages 2–11, which ultimately could lead to a label expansion should the therapy be deemed safe and effective in pediatric patients.
APeX-P is recruiting children with HAE at sites in Canada, Austria, France, Israel, and Spain. Additional sites in Germany, the U.K., Italy, Romania, and Poland are expected to open.
Marketing in other regions
BioCryst has partnerships with a number of other companies to market the therapy in various geographical regions worldwide.
In Latin America, including Chile, Pint Pharma will be responsible for obtaining and maintaining any marketing authorizations and for commercializing the therapy in that region.
Torii Pharmaceutical is marketing the treatment in Japan. Swiss company Swixx BioPharma will commercialize Orladeyo in central and Eastern Europe.
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