Investing.com -- BioCryst Pharmaceuticals (NASDAQ:BCRX) stock rose 4% after the company announced FDA approval of ORLADEYO oral pellets for prophylactic treatment of hereditary angioedema (HAE) in children aged 2 to under 12 years.
The approval makes ORLADEYO the first and only oral prophylactic treatment option for young HAE patients. Until now, children under 12 with the condition only had access to treatments administered intravenously or through subcutaneous injection.
The FDA’s decision was supported by interim data from the APeX-P clinical trial, which showed the treatment was well tolerated with consistent safety profiles and demonstrated sustained reductions in monthly attack rates. The most common side effect reported was nasopharyngitis.
"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," said Anthony J. Castaldo, CEO of the U.S. Hereditary Angioedema Association.
The new pellet formulation is designed specifically for children, allowing for easy administration by pouring directly into the mouth or sprinkling over soft food. BioCryst noted that HAE often presents during childhood, with approximately 40% of children experiencing their first attack by age 5.
The capsule formulation of ORLADEYO was initially approved in December 2020 for patients 12 years and older and is now approved in more than 45 countries. According to the company, the treatment has been prescribed to more than 3,500 patients in the U.S. to date.
BioCryst has also filed for approval of the oral pellets formulation in Europe.
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