First oral treatment to prevent HAE attacks in children older than two approved by US FDA

The US FDA (Food and Drug Administration) has approved the use of an oral pellet formulation of the HAE treatment berotralstat (also referred to by its brand name, Orladeyo) to prevent HAE attacks in children aged two and above (2 to <12).

Berotralstat is the first and only targeted oral prophylactic therapy for HAE patients over the age of two. The new pellet formulation is sprinkle-like in appearance and size, and can be poured directly into the mouth with water or milk, or sprinkled over a spoonful of soft, non-acidic food.

Anthony J Castaldo, CEO and Chairman of the US Hereditary Angioedema Association (HAEA), said: “Today’s pediatric approval of Orladeyo 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.”

Charlie Gayer, President & Chief Commercial Officer of BioCryst, said: “We are excited to make Orladeyo oral pellets available to children living with HAE. 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.”

Jon Stonehouse, Chief Executive Officer of BioCryst, said: “We couldn’t be more excited to bring this treatment option to these kids and their caregivers,” said. “Orladeyo has been prescribed to more than 3,500 patients in the US 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. Thank you to the patients and caregivers, investigators, advocates, and employees who helped make this significant HAE treatment milestone possible.”

BioCryst has filed applications for the use of berotralstat oral pellets in patients with HAE aged 2 to <12 years with the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency. Additional regulatory filings are planned in other global territories, including Canada.

(Source: BioCryst)