Oral HAE treatment Orladeyo now available to patients in Portugal
Country's regulatory agency recommends therapy's use to prevent swelling
The government agency in Portugal that regulates human medicines, Infarmed, has recommended Orladeyo (berotralstat) for the routine prevention of swelling attacks in people with hereditary angioedema (HAE), ages 12 and older.
With this positive recommendation, HAE patients in the country will now be able to access Orladeyo treatment. According to the therapy’s developer Biocryst Pharmaceuticals, this makes Orladeyo the first ever once-daily prophylactic, or preventive, treatment for HAE to become available in Portugal country.
“The positive Infarmed 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 Portugal,” Charlie Gayer, chief commercial officer of Biocryst, said in a company press release.
Orladeyo now licensed in 44 countries worldwide, per Biocryst
HAE is a genetic disorder marked by abnormally high levels of a signaling molecule called bradykinin, which leads to the swelling attacks that characterize the disease. Orladeyo works to block the activity of kallikrein, the enzyme that produces bradykinin. By blocking kallikrein, the therapy aims to lower bradykinin levels, thereby reducing the risk of swelling attacks.
Orladeyo was approved in the European Union in 2021 as a prophylactic treatment for HAE for patients ages 12 and older. It had been similarly approved in the U.S. in late 2020, and more recently has won regulatory approvals in other countries, such as Argentina, Brazil, Chile, Mexico, and Peru. According to Biocryst, the therapy is now licensed in 44 countries worldwide.
Approvals of Orladeyo were based in large part on data from a Phase 3 clinical trial called APeX-2 (NCT03485911), which tested the therapy against a placebo in more than 100 people with HAE.
Over the course of the study’s six-month initial phase, participants given the placebo experienced more than two swelling attacks per month, on average. Those on Orladeyo had slightly more than one attack per month — a statistically significant difference.
Long-term data from APeX-2 indicated that Orladeyo’s effectiveness was consistent for as long as two years. Data also have suggested that patients switching to the oral therapy from previously-available injection treatments generally expressed high satisfaction.
Consistent with data from clinical trials, real-world data from the U.S. has suggested that Orladeyo can limit swelling attacks and reduce hospitalizations, and thus healthcare use.
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