FDA extends Orladeyo approval to children as young as 2
Treatment becomes only oral prophylactic OKd for kids 2-11
The U.S. Food and Drug Administration (FDA) expanded the approval of Orladeyo (berotralstat), making it the only oral treatment approved in the U.S. as a prophylactic (preventive) therapy for children with hereditary angioedema (HAE) ages 2-11.
The new authorization extends the FDA’s 2020 approval of Orladeyo as a prophylactic therapy for HAE patients ages 12 and older. Developer Biocryst Pharmaceuticals had filed an application for approval in younger children.
“As we mark five years since Orladeyo first transformed care for those with HAE ages 12 and older, this approval extends the benefits of oral prophylactic therapy to a vulnerable and important part of the HAE community, children ages 2 to less than 12. We couldn’t be more excited to bring this treatment option to these kids and their caregivers,” Jon Stonehouse, CEO of Biocryst, said in a company press release.
‘More freedom to live a normal life’
“Orladeyo has been prescribed to more than 3,500 patients in the U.S. to date, and we’re honored to now bring an oral pellet formulation to children and their caregivers, answering the community’s heartfelt call for a long-term prophylactic treatment option that meets the unique needs of children,” Stonehouse said.
Biocryst has also submitted applications seeking to expand approval of Orladeyo for younger children with HAE in Japan and in Europe, where the company recently sold the rights to the therapy. A similar application in Canada is in the works.
“We are excited to make Orladeyo oral pellets available to children living with HAE,” said Charlie Gayer, president and chief commercial officer of Biocryst. “The use of prophylactic therapy has been limited among younger children compared to adults, in part because of the lack of appropriate, less burdensome treatment options. Orladeyo oral pellets have the potential to change how a new generation of children with HAE and their caregivers manage this condition, giving them more freedom to live a normal life.”
Anthony J. Castaldo, CEO and chairman of the U.S. Hereditary Angioedema Association, said the expanded FDA approval “offers a welcome oral preventive choice for children living with HAE and provides families and clinicians with an important option for shared decision-making that matches treatment with patient needs.”
HAE is a genetic disorder characterized by episodes of swelling due to the overproduction of a signaling molecule called bradykinin. Orladeyo is designed to inhibit kallikrein, an enzyme required for bradykinin production. The oral therapy aims to keep bradykinin levels low and prevent swelling. Although other prophylactic HAE therapies are available, they’re given by injection or infusion, which is generally considered less convenient for patients and their families than oral meds.
“Pediatric patients living with HAE can experience significant burdens, not only from the disease itself but also from its treatments, affecting their physical, psychosocial, and developmental well-being,” said Raffi Tachdjian, MD, a professor at the University of California Los Angeles. “This new oral pellet formulation of Orladeyo provides a more easily administered long-term-prophylactic option to help children with HAE better manage their disease, especially when the experience of infusions or injections is not ideal.”
Biocryst’s application seeking approval of Orladeyo for kids younger than 12 was based primarily on findings from an ongoing Phase 3 clinical trial called APeX-P (NCT05453968), which is evaluating the daily oral therapy in 29 children with HAE ages 2 to 11. Interim data showed that the average rate of swelling attacks fell from 1.5 per month to 0.3 per month in the year following the start of Orladeyo treatment.
Safety data from the pediatric study were generally consistent with findings from older patients. Common side effects of Orladeyo include digestive complaints and back and/or abdominal pain. The therapy’s prescribing information notes that it should not be given to children younger than 12 who have severe kidney impairment, and it’s not recommended for patients of any age with end-stage kidney disease.
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