NICE recommends BioCryst’s ORLADEYO®, an oral, once-daily therapy, to prevent recurrent attacks of hereditary angioedema in patients 12 years and older
RESEARCH TRIANGLE PARK, N.C. – September 16, 2021– BioCryst Pharmaceuticals today announced that the National Institute for Health and Care Excellence (NICE) has issued a recommendation for ORLADEYO® (berotralstat), that will provide hereditary angioedema (HAE) patients in England, Wales and Northern Ireland access to the first licensed, oral, once-daily therapy for routine prevention of recurrent HAE attacks in eligible adults and adolescents aged 12 years and older experiencing at least two attacks per month.1,2
“HAE is a very rare genetic condition which is painful and debilitating and can be fatal if left untreated. The unpredictability of the condition severely affects quality of life for patients and their families,” said Laura Szutowicz, chief executive officer of HAE UK. “HAE UK welcomes the NICE decision on berotralstat which means that eligible patients and clinicians have another choice of treatment for this lifelong condition.”
NICE guidance was based on findings from the Phase 3 APeX-2 trial, in which berotralstat 150mg dose (n=40) met its primary endpoint, significantly reducing HAE attacks vs. placebo at 24 weeks.* Data presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2021 from the long-term extension of the APeX-2 study at 96 weeks, showed that patients taking berotralstat 150 mg demonstrated an 80 percent average reduction in mean attack rate per month during weeks 25-96 compared to baseline (n=21). Once-daily berotralstat was generally well tolerated throughout the APeX-2 study.3 The most frequently reported adverse reactions in patients receiving berotralstat compared with placebo were abdominal pain, diarrhoea, and headaches. Gastrointestinal symptoms were predominantly mild to moderate, and mostly subsided after the first month of treatment.1
“HAE is a very rare genetic condition which is painful and debilitating and can be fatal if left untreated. The unpredictability of the condition severely affects quality of life for patients and their families.”
Laura Szutowicz, chief executive officer of HAE UK
“The impact of HAE on patients goes beyond the potentially life-threatening swellings – it can also negatively affect self-esteem and have a long-term impact on quality of life,” said Dr Sorena Kiani, consultant immunologist at Barts Health NHS Trust. “The NICE recommendation of berotralstat is great news for clinicians and eligible patients who now have access to the first oral preventive treatment for HAE that could significantly reduce the number of attacks and may improve quality of life in many patients with the condition.”
*In APeX-2, berotralstat met its primary endpoint for the 150mg dose, by reducing the frequency of HAE attacks by 44% (n=40) when compared with placebo (n=39) at 24 weeks (rate per 28 days: 1.31 vs 2.35; p<0.001).
“We are excited for this recommendation from NICE which provides eligible HAE patients in the UK access to the first oral, once-daily treatment that could help achieve symptom control and provide relief from the debilitating symptoms of HAE. The positive NICE recommendation also widens access for patients to modern prophylaxis in the UK.”
Charlie Gayer, chief commercial officer of BioCryst.
The decision follows European Commission (EC) marketing authorisation of berotralstat in April 2021 and authorisation from UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in May 2021.1,4 A decision from the Scottish Medicines Consortium (SMC) for use of berotralstat for HAE patients in Scotland is anticipated in the first half of 2022.
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About ORLADEYO® (berotralstat)
”ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
Please refer to the Summary of Product Characteristics for more:
https://mhraproducts4853.blob.core.windows.net/docs/92f95892739349fc24b9a9355a3d4e0f7205abe0About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily berotralstat has been granted a licence in the United States, the European Union, Japan, the United Arab Emirates and the United Kingdom for the prevention of recurrent HAE attacks in adults and adolescents aged 12 years and older. ®
- Summary of Product Characteristics for Great Britain. ORLADEYO TM (2021) 150 mg berotralstat hard capsule.
- Available from: https://mhraproducts4853.blob.core.windows.net/docs/92f95892739349fc24b9a9355a3d4e0f7205abe0. Last Accessed: September 2021 ® ®
- NICE Final Appraisal Determination for ORLADEYO. Available from: https://www.nice.org.uk/guidance/gid-ta10650/documents/final-appraisal-determination-document. Last Accessed: September 2021.
- Kiani. S. Durable Reduction in Hereditary Angioedema (HAE) Attack Rates with Berotralstat Over 24 Months: Results from the Phase 3 APeX-2 Study. Abstract presented at European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2021.
- European Medicines Agency. Orladeyo. Available from:
- https://www.ema.europa.eu/en/medicines/human/EPAR/orladeyo#authorisation-details-section. Last accessed: September 2021. ®
Job code: UK.HAE.00050
Date of preparation: September 2021