Preventive angioedema drug now OK’d in Japan for kids as young as 2
Regulators expand use of approved oral prophylaxis for HAE swelling attacks
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Biocryst Pharmaceuticals‘ once-daily oral therapy Orladeyo (berotralstat) is now approved in Japan to prevent swelling attacks in children with hereditary angioedema (HAE) as young as 2 years old.
Japan’s Ministry of Health, Labor and Welfare (MHLW) first approved Orladeyo in 2021 as a treatment for HAE patients ages 12 and older. Now, the MHLW has extended its approval to also cover children ages 2 to 11.
“The approval of Orladeyo for children in Japan marks an important step toward our goal of bringing a once-daily oral treatment to people living with HAE around the world,” Charlie Gayer, president and CEO of Biocryst, said in a company press release announcing the decision.
Biocryst said it plans to commercially launch Orladeyo in Japan for children with HAE after it completes the reimbursement pricing process with the Japanese National Health Insurance System. Orladeyo will be sold in the Asian nation via Biocryst’s representative partner, Orphanpacific.
“We’re proud to advance an oral prophylactic option in Japan that can help reduce treatment burden for children and provide greater convenience for families managing this lifelong disease,” Gayer said.
A genetic disorder, HAE is marked by bouts of swelling that can be painful, disfiguring, and in some cases, life-threatening. Swelling in HAE is driven by excessive activity of a signaling molecule called bradykinin, which is produced by an enzyme known as kallikrein. Orladeyo works by inhibiting kallikrein activity, thereby reducing bradykinin levels and ultimately preventing swelling.
New Orladeyo approval based on strong trial results
In children younger than 12, the daily oral therapy is given as a pellet or granule formulation, which can be swallowed directly or sprinkled over a tablespoon of nonacidic soft food, such as bananas or mashed potatoes. In older children and adults, Orladeyo is given as a daily capsule.
Approval of Orladeyo for young children in Japan comes less than a year after a similar decision in the U.S. In December, the U.S. Food and Drug Administration extended approval of Orladeyo for use in children with HAE as young as 2.
Both approvals were based on data from APeX-P (NCT05453968), an open-label clinical trial testing prophylactic (preventive) treatment with Orladeyo in more than two dozen youngsters, ages 2 to 11, with HAE. Most participants were free from swelling attacks for more than one year on Orladeyo, and no new safety issues were reported.
Common side effects of the approved treatment are abdominal pain, back pain, and digestive complaints.
Biocryst has submitted applications seeking approval of Orladeyo for children as young as 2 in Canada and Europe. As of now, Orladeyo is approved for HAE patients 12 and older in those countries. The company said it is also working on similar applications in other markets.
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