Biocryst seeks FDA OK of Orladeyo for HAE patients as young as 2
Company seeking similar approvals in Europe, Japan, Canada
Biocryst Pharmaceuticals has submitted an application asking the U.S. Food and Drug Administration (FDA) to extend the approval of Orladeyo (berotralstat) to children with hereditary angioedema (HAE) as young as 2 years.
The application specifically seeks the approval of an oral granule formulation of the therapy for children ages 2-11. If approved, Orladeyo would become the first targeted oral preventive therapy to be approved for children with HAE.
The application is a “move closer to delivering an urgently needed oral prophylactic therapy to children with HAE,” Helen Thackray, MD, Biocryst’s chief research and development officer, said in a company press release.
Biocryst also stated it is planning to submit similar applications in other worldwide markets, including Europe, Japan and Canada.
Orladeyo is an oral preventive therapy used to reduce the frequency of swelling attacks in HAE. It is currently approved in the U.S. and other countries for people, 12 and older, with HAE . The medication works by blocking the activity of kallikrein, an enzyme needed to make a signaling molecule called bradykinin, which is excessively produced in HAE patients and is believed to act as the trigger for the swelling attacks that characterize the disease.
Current approved HAE therapies for young children are given by injection
While there are therapies currently available to prevent or lower the frequency of swelling attacks in children with HAE, all of them are given through injections.
“Despite significant innovation in HAE prophylaxis for adults, there is still high unmet need in children,” the company stated in a corporate update presentation. In this context, the submission of the new application to the FDA “positions Orladeyo to be the market leading prophylaxis for children.”
An ongoing Phase 3 clinical trial called APeX-P (NCT05453968) is assessing the safety and pharmacological properties of once-daily Orladeyo oral granules in 29 children with HAE, ages 2-11.
Recent data from APeX-P showed Orladeyo was generally well tolerated in these young children. No new or unexpected safety issues and no serious side effects related to Orladeyo were reported. Common side effects of the therapy in young children included headache, upper respiratory infections, and the common cold.
Available data also indicated Orladeyo was able to reduce the rates of swelling attacks for children in the study. The mean rate of monthly attacks decreased from 1.5 attacks per month during an initial period where children were on standard-of-care treatment to 0.3 attacks per month after one year of treatment with Orladeyo.
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