Paediatric Medicines2 November 2026

A pellet built for young children reaches adults with hereditary angioedema

The FDA has extended BioCryst's Orladeyo oral pellets to patients aged 12 and over, ten months after approving the formulation for younger children. It reverses the usual order of paediatric development.

By NextGenHealth Editorial Team2 min read

A pile of white pills on a blue table — Photo: Hal Gatewood / Unsplash

In brief

BioCryst's Orladeyo oral pellets, first approved for children aged two to 11, now have a 150 mg FDA-approved dose for patients aged 12 and over.

BioCryst announced FDA approval of a 150 mg once-daily berotralstat pellet for patients aged 12 and over on 21 October 2026.
The supplemental application relied on manufacturing data and a safety and tolerability comparison with the capsule.
Pellets were first approved for children aged two to under 12 on 12 December 2025.

BioCryst Pharmaceuticals announced on 21 October 2026 that the FDA had approved a 150 mg once-daily oral pellet of Orladeyo (berotralstat) to prevent attacks of hereditary angioedema (HAE) in patients aged 12 and over. Berotralstat now has two approved oral formulations for that age group, a capsule and a pellet.

Paediatric formulations usually follow adult products, often years later. Here the sequence ran the other way. The FDA approved pellets for children aged two to under 12 on 12 December 2025, and the capsule had been available from age 12 since December 2020.

How the approval was built

The decision came through a supplemental application, based on chemistry, manufacturing and controls information and data on safety and tolerability comparing the pellet with the capsule. HCPLive reported that the company gave no new efficacy data for the pellet at this dose. Efficacy rests on the capsule, which across two studies reduced the mean attack rate by 91% at 96 weeks in patients aged 12 and over, while rescue medication use fell by 88%.

Charlie Gayer, BioCryst's chief executive, said the approval "strengthens our ability to provide personalized treatment choice". Any commercial gain will depend on how many older patients prefer pellets to a capsule.

A formulation developed for young children has become an extra option for adults.

The younger group

The clinical need is sharpest in younger children. According to BioCryst, about 40% of children with HAE have their first attack by the age of five. Pellets come in unit-dose packets of 72, 96, 108 or 132 mg, dosed by weight, for those aged two to 11.

Interim data from the APeX-P trial, presented at the EAACI congress in Istanbul in June 2026, covered 29 patients aged two to under 12 who weighed at least 12 kg and had C1-inhibitor deficiency. Seven took capsules and 22 took pellets. The rate of attacks needing on-demand treatment was 0.169 a month on berotralstat, against 0.691 in the standard-of-care period. That is an uncontrolled comparison in a small group, so it offers reassurance on direction more than a precise effect size.

Safety and labelling

The prescribing information carries age-specific limits. Use should be avoided in children aged two to 11 with moderate or severe hepatic impairment, and the safety and effectiveness of the drug have not been established below the age of two. Increases in the QTc interval have been seen in adults at doses above 150 mg once daily. Berotralstat is for prophylaxis and is not a treatment for acute attacks.

Beyond the US

BioCryst said in December 2025 that it had filed the paediatric pellets with the European Medicines Agency and Japan's PMDA, and that a Canadian filing was planned. For companies with rare disease products, the case points to a practical lesson: a formulation designed for the youngest patients can serve the whole label, and can get there on a relatively modest regulatory package.

Hereditary angioedema
Berotralstat
Formulations
BioCryst

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