FDA approves BioCryst’s Pellet Formulation of Orladeyo for HAE

Rare Daily Staff

The U.S. Food and Drug Administration has approved a pellet formulation of BioCryst Pharmaceuticals’ Orladeyo for adults and adolescents with hereditary angioedema, a rare and potentially life-threatening condition.

The approval gives patients who have difficulty swallowing capsules another way to take the daily preventive medicine. Orladeyo capsules were first approved in the United States in December 2020 for patients ages 12 and older. The latest approval expands the options for administering the drug to this age group. The FDA previously approved a pellet formulation for children ages 2 through 11 in December 2025.

The pellets resemble small sprinkles. Patients can pour them directly into their mouths and immediately swallow them with water or milk, or sprinkle them over a spoonful of soft, non-acidic food.

Hereditary angioedema (HAE) is a genetic disorder that causes recurring attacks of swelling in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. These attacks can be debilitating and painful. Attacks that obstruct the airways are potentially life-threatening because of the risk of asphyxiation.

Orladeyo, also known as berotralstat, works by reducing the activity of plasma kallikrein to prevent hereditary angioedema attacks. It is not intended to treat an attack already underway. According to BioCryst, it is the only targeted oral preventive treatment with options for hereditary angioedema patients as young as 2.

BioCryst said the FDA’s decision was based on manufacturing and quality-control information, along with data demonstrating the safety and tolerability of the pellets compared with the capsules.

“The approval of Orladeyo pellets for adults and adolescents strengthens our ability to provide personalized treatment choice,” said Charlie Gayer, BioCryst’s president and CEO.

Photo: Charlie Gayer, BioCryst’s president and CEO