FDA to review Orladeyo application for young children with HAE
Therapy would be first targeted oral treatment for swelling in children under 12
The U.S. Food and Drug Administration (FDA) has accepted Biocryst Pharmaceuticals’ application to approve an oral granule formulation of Orladeyo (berotralstat) for children with hereditary angioedema (HAE) as young as 2.
The FDA granted the application priority review, which shortens the process from the standard 10 months to within six months. If approved, Orladeyo would be the first targeted oral treatment to prevent swelling attacks in children with HAE under age 12. A decision is expected by Sept. 12, according to Biocryst.
“We are excited to take another step closer to bringing Orladeyo to younger pediatric patients with HAE,” Jon Stonehouse, president and CEO of Biocryst, said in a company press release. “We consistently hear from patients, caregivers, and physicians about their desire for a more convenient therapeutic option to treat young children with HAE, and we now may have the opportunity to bring this to them later this year.”
The company has filed a similar application to European regulatory authorities and plans to submit requests in other regions, including Canada and Japan.
“If approved, we believe this oral granule formulation of Orladeyo could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays,” said Helen Thackray, MD, chief research and development officer of Biocryst.
How does Orladeyo work in HAE?
A genetic condition, HAE is marked by the overproduction of bradykinin, a signaling molecule that regulates blood pressure and inflammation by widening blood vessels. When there’s too much of it, fluids leak from blood vessels into nearby tissues, leading to sudden swelling attacks.
Orladeyo is an oral therapy approved in the U.S. and other countries to prevent swelling attacks in HAE patients, ages 12 and older. It blocks kallikrein, an enzyme needed to make bradykinin, thereby lowering bradykinin levels and preventing swelling.
The application seeking Orladeyo’s approval for younger children was based on interim data from the ongoing Phase 3 APeX-P clinical trial (NCT05453968), which is assessing the pharmacological properties and safety of once-daily Orladeyo oral granules in 29 children with HAE, ages 2-11.
Interim data showed the formulation reduced the mean rate of swelling attacks from 1.5 per month with standard of care to 0.3 a month after a year of treatment.
“As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of HAE symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far,” Thackray said.
The therapy has been determined to be safe and well tolerated, with no new safety issues or serious side effects related to treatment. Common side effects included upper respiratory infections, the common cold, and headaches.
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