FDA Approves Berotralstat as First Oral Hereditary Angioedema Prophylaxis


The indication is based on 48-week results showing significant benefit in reduced HAE attacks among pediatric and adult patients.


The US Food and Drug Administration (FDA) has approved berotralstat (ORLADEYO) for the prevention of hereditary angioedema (HAE) attacks in adults and pediatrics aged 12 years and older.

The once-daily oral therapy from BioCryst Pharmaceuticals becomes the first of its kind indicated for HAE attack prophylaxis.

Pivotal phase 3 data from the APeX-2 supporting berotralstat for the new indication showed a 150 mg dose of the therapy was associated with a significantly reduced risk of HAE attack at 24 weeks, which was sustained through the full 48-week treatment period.

Among patients treated with berotralstat for the full 48 weeks, mean monthly HAE attack decreased from 2.9 to 1.0. In a long-term open-label assessment of the same patient population, investigators observed a decrease to just 0.8 attacks monthly.

In both studies, the therapy was associated with known safety and tolerability, with the most frequently reported treatment-related adverse events being gastrointestinal reactions.

The historic indication for berotralstat provides confidence in clinicians and patients seeking to make progress in reduced HAE attack risks—without the burden of infusion or injection therapy, APeX-2 trial investigator Marc Riedl, MD, professor of medicine and clinical director at the US Hereditary Angiodema Association Center at the University of California, San Diego, said.

“Patients and physicians acknowledge that HAE treatments can add a burden to patients’ lives,” Riedl said in a statement. “With this new treatment option, physicians and patients can continue to have collaborative discussions to choose the treatment that meets each patient’s needs, life circumstances and preferences.”

Jon Stonehouse, president and chief executive officer of BioCryst, echoed sentiment on the significance of the approval.

“The FDA approval of ORLADEYO fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks,” he said. “We will stay focused on enabling access and providing personalized support to HAE patients and physicians.”

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