The U.S. Food and Drug Administration approved BioCryst Pharmaceuticals’ oral pellet formulation of ORLADEYO (berotralstat) for prophylactic use in pediatric patients with hereditary angioedema (HAE) ages 2 to under 12 years, expanding the drug’s indication from adults and adolescents to a younger, previously untreated segment of the HAE market.
The approval was supported by the APeX‑P clinical trial, which demonstrated early and sustained reductions in monthly attack rates and a favorable safety profile, with most patients reporting the pellet form as well tolerated and easier to administer than injections or infusions.
In Q3 2025, BioCryst reported net revenue of $159.1 million, a 37% year‑over‑year increase, and raised its full‑year 2025 revenue guidance to $590 million–$600 million. The pediatric approval is expected to add $100 million–$150 million in peak annual sales once the product is launched, reflecting the sizable unmet need in the 2‑to‑12‑year age group and the pellet’s improved palatability and dosing convenience.
CEO Jon Stonehouse highlighted the sale of BioCryst’s European ORLADEYO business as a key step in strengthening the company’s balance sheet and reducing debt, while President and Chief Commercial Officer Charlie Gayer emphasized that making ORLADEYO oral pellets available to children will transform HAE management by eliminating the need for injections or infusions in this population.
Strategically, the approval fits into BioCryst’s broader plan to expand its HAE portfolio. The company has entered a definitive agreement to acquire Astria Therapeutics, which will add the late‑stage injectable therapy navenibart to its pipeline, and the European business sale has already helped improve cash flow and reduce leverage.
The HAE market remains competitive, with established products such as Takeda’s Takhzyro and newer agents like garadacimab‑gxii, sebetralstat, and donidalorsen. ORLADEYO’s oral administration and the new pellet form give it a distinct advantage in pediatric patients, where ease of use is a critical factor in adherence and quality of life.
Financially, BioCryst’s Q3 2025 net income of $12.9 million (versus a $14.0 million loss in Q3 2024) signals a turnaround, but the company’s debt‑to‑equity ratio remains negative and its Altman Z‑Score of –1.95 indicates a distress zone. Management expects to achieve positive cash flow and quarterly earnings per share profitability in the second half of 2025, driven by the new pediatric indication and the ongoing commercialization of the adult product.
The FDA approval not only expands BioCryst’s addressable market but also reinforces its position as a leader in rare‑disease therapeutics, supporting the company’s long‑term growth strategy while highlighting the need to manage leverage and continue investing in its pipeline.
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