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BioCryst new long-term study data and an independent 6 month follow up of patients enrolled in the UK Early Access to Medicines Scheme (EAMS) demonstrates the long-term impact of prophylactic Orladeyo® (berotralstat) on HAE

BioCryst new long-term study data and an independent 6 month follow up of patients enrolled in the EAMS

RESEARCH TRIANGLE PARK, N.C. – July 1, 2022 BioCryst Pharmaceuticals is presenting new 96-week data from the open-label global randomised Phase III APeX-2 trial, demonstrating that patients over 12 years of age with Hereditary Angioedema (HAE) receiving Orladeyo® (berotralstat) 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 2022.1,2

“The APeX-2 results demonstrate berotralstat’s long-term efficacy as a prophylactic treatment for HAE attacks,” said Luke Robinson, VP, general manager, UK, Ireland & Nordics BioCryst Pharmaceuticals. “These data reaffirm BioCryst’s ongoing commitment to pursue solutions to help improve the lives of people living with rare diseases like HAE.”

The APeX-2 study analysis assessed the efficacy and safety of 150 mg berotralstat in reducing the number of attacks per month requiring treatment with injectable on-demand medication, over 96 weeks of treatment from baseline.1 The data showed that patients with HAE who completed 96 weeks of treatment with 150 mg of oral berotralstat (n=21) saw: 1

  • 87 percent reduction in mean number of HAE attacks requiring injectable treatment per month, compared to baseline [the mean number of treated attacks was 2.2(0.42) attacks/month at baseline, which declined to 0.9(0.34) attacks/month at Week 24, 0.6(0.25) attacks/month at Week 48, and 0.4(0.15) attacks/month at Week 96]. 
  • The overall use of on-demand injectable medication declined over time, with a decrease in the number of doses per month of 88 percent by week 96 [at baseline a mean of 2.8(0.54) doses/month of on-demand medication was used to treat attacks. This declined to 1.1(0.38) doses/month at Week 24, 0.8(0.31) doses/month at Week 48, and 0.4(0.18) doses/month at Week 96]. 

“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.”

The APeX-2 trial findings, showing reduced attack rate and suggested improved overall disease control, were supported by publication of an independent UK analysis of real-world experience of berotralstat. Preliminary results from 54 patients across 12 UK centres that initiated berotralstat as part of the Early Access to Medicines Scheme (EAMS) between November 2020 to April 2021 showed:2 

  • A significant 52 percent reduction in HAE attack rate per month over the first three months of treatment and a 65 percent reduction between months three and six of treatment compared to the three months prior to starting berotralstat (P<0.0001, n=28-33)
  • Significant improvement in the severity of those attacks that did occur. Patient-rated severity (where 1 = very mild, 5= very severe) declined from a baseline of 3.47+/-0.79 in the 3 months prior to treatment to 2.50+/-1.21 during months 1 – 3 and to 2.34+/-1.17 during months 4 – 6 (P<0.0001, n=38-49) 
  • Significant improvement in Angioedema Control Test Score (AECT)* from 4.93±3.42 attacks over 3 months prior to initiation to 9.79±4.53 and 11.03±3.14 during months 1-3 and 4-6 respectively (P<0.0001, n=32-43)

“This independent multi-centre evidence from the UK is in keeping with the results seen in the Phase 3 clinical trials and suggests that berotralstat is an effective treatment option in real world clinical practice,” said Dr George Stanley, Medical Director BioCryst UK Ltd. “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”

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/airway, or abdominal areas.3,4 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.4

Berotralstat is indicated for the routine prevention of recurrent HAE attacks in adult and adolescent patients aged 12 years and older in the European Union (EU) and United Kingdom.5 Berotralstat is not intended for treatment of acute HAE attacks, individualised treatment should be initiated with an approved rescue medicinal product.5

Please refer to the Summary of Product Characteristics for more: 

*AECT is a simple validated patient reported outcome tool for routine use in the clinic to assess disease control in HAE and guide treatment decisions. A cutoff value of 10 or more points is used to identify patients with well-controlled HAE.6

About 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 Orladeyo® (berotralstat) is approved 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 paediatric patients 12 years and older. 


[1]Smith M. A., Magerl M., Desai B. et al. 96 Weeks of Treatment with Berotralstat Consistently Decreases the Use of Injectable On-Demand Medication to Treat Hereditary Angioedema (HAE) Attacks: Analysis from APeX-2. Abstract presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress in Prague, Czech Republic, 1-3 July 2022. 

[2]Ahuja M., Dorr A., Bode E., et al. Berotralstat for the prophylaxis of hereditary angioedema – a national survey of patient outcomes. LB Abstract presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress in Prague, Czech Republic, 1-3 July 2022

[3]Bygum A, et al. The hereditary angioedema burden of illness study in Europe (HAE-BOIS-Europe): background and methodology. BMC Dermatol. 2012; 12: 4. doi: 10.1186/1471-5945-12-4

[4]Bork K, Anderson JT, Caballero T et al. Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report. Allergy Asthma Clin Immunol. 2021 Apr 19;17(1):40.

[5]EMA. Orladeyo Summary of Product characteristics. Available at:  Last accessed: June 2022. 

[6]Weller K., Donoso T., Magerl M., et al.  Validation of the Angioedema Control Test (AECT)—A Patient-Reported Outcome Instrument for Assessing Angioedema Control. JAIC-In practice. (2020).

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