BioCryst’s Orladeyo wins expanded FDA approval

The US Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for the use of an oral pellet formulation of once-daily Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.

The drug is part of North Carolina, USA-based biotech BioCryst Pharmaceuticals (Nasdaq: BCRX) HAE portfolio, which the company expects to expand via the planned acquisition of Astria Therapeutics (Nasdaq: ATXS), along with its prophylaxis candidate navenibart, that was announced in October.

“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 Castaldo, chief executive and chairman of the US Hereditary Angioedema Association (HAEA).