Long-Term Impact of Prophylactic Orladeyo on Hereditary Angioedema

pharmafile | July 4, 2022 | News story | Sales and Marketing  

BioCryst Pharmaceuticals have shared new 96-week data from the open-label global randomised Phase III Apex-2 trial, and an independent six month follow-up, of patients enrolled in the UK Early Access to Medicines Scheme (EAMS) for Prophylactic Orladeyo®.

The prophylactic, Orladeyo (berotralstat), is for the treatment of patients over 12 years of age with Hereditary Angioedema (HAE). Patients in the Phase III trial given Orladeyo experienced fewer attacks that required treatment with injectable on-demand medication, and used less on-demand medication (doses/month) to treat attacks from baseline.

These data are being presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress in Prague, Czech Republic, 1-3 July 2022.

“The unpredictable need for on-demand injectables to control HAE attacks can have an impact on a patient’s day-to-day functioning and overall quality of life,” said Dr Sorena Kiani, consultant immunologist at Barts Health NHS Trust. “The APeX-2 study shows that a 150 mg dose of berotralstat taken as an oral once daily therapy can reduce the number of HAE attacks per month and reduce the use of on-demand injectables. This allows patients to have more needle-free days per month which is likely to contribute to improved quality of life.”

HAE is a rare genetic condition characterised by swelling attacks in the skin or mucous membranes, resulting in painful swelling in the extremities, face, throat or airway, or abdominal areas. Swelling onset can be spontaneous, or result from trauma or other specific triggers. Due to potential swelling of the throat, HAE carries the risk of asphyxiation and can be potentially life-threatening.

Ana Ovey

Dr George Stanley, Medical Director BioCryst UK Ltd, commented: “EAMS enables patients with significant unmet needs to gain early access to innovative treatments. Through participation in EAMS, HAE specialist centres have been able to generate valuable real-world evidence for berotralstat which contributed to positive Health Technology Appraisal by NICE and SMC.”

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