RESEARCH TRIANGLE PARK, N.C. – BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the European Commission (EC) has approved oral, once-daily ORLADEYO™ (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
“As the first targeted oral prophylactic therapy approved in Europe, ORLADEYO represents a major advance in treatment for HAE patients who have been waiting for a preventive therapy. Physicians will be delighted to discuss this new option with their patients,” said Emel Aygören-Pürsün, M.D., head of the HAE Center at the University Hospital in Frankfurt.
“ORLADEYO offers people with HAE in Europe and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents a vitally important and most welcome step in making more treatment options available,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE.
The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.
BioCryst has its European commercial team in place and expects to launch ORLADEYO this quarter in Germany, with launches in other European markets to follow. HAE patients in France currently have access to ORLADEYO through an Autorisation Temporaire d’Utilisation de cohorte (cohort ATU).
In the United Kingdom, HAE patients also currently have access to ORLADEYO through an approved early access to medicines scheme (EAMS). A marketing authorization application (MAA) has been submitted to the Medicines and Healthcare products Regulatory Agency (MHRA). Under the new European Commission Decision Reliance Procedure, the MHRA will aim to complete the review of the UK MAA as soon as possible following the EC approval decision.
“Most European HAE patients today treat their disease with on-demand therapy or androgens and we believe the approval of oral, once-daily ORLADEYO provides an exciting new opportunity for these patients to reduce their burden of therapy by moving to prophylaxis with ORLADEYO,” said Jon Stonehouse, president and chief executive officer of BioCryst.
“We saw tremendous enthusiasm and participation from European HAE patients in our clinical trials and we have invested in an experienced European commercial team that is excited to bring ORLADEYO to HAE patients across Europe,” he added.
In the pivotal Phase 3 APeX-2 trial, ORLADEYO significantly reduced attacks at 24 weeks, and this reduction was sustained through 48 weeks. HAE patients who completed 48 weeks of treatment (150 mg) saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy. In the long-term open label APeX-S trial, patients completing 48 weeks of therapy (150 mg) had a mean attack rate of 0.8 attacks per month.
ORLADEYO was safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time and typically self-resolved.
HAE patients note a significant treatment burden associated with existing prophylactic therapy. In addition to reducing HAE attack rate, data from APeX-2 show that patients reported meaningful improvements in both quality of life, overall patient-reported satisfaction, and significant reductions in their monthly use of standard of care on-demand medicine, while taking oral, once-daily ORLADEYO (150 mg). 1,2
About ORLADEYO™ (berotralstat)
ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
The full European Summary of Product Characteristics (SMPC) for ORLADEYO will be available from the European Medicines Association website at www.ema.europa.eu.
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, European Union and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company’s website at www.biocryst.com.
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1 Johnston, D.T., et al. Berotralstat Improves Patient-Reported Quality of Life Through 48 Weeks in the Phase 3 APeX-2 Trial; Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology; Poster 154, Nov (2020)
2 Jacobs, J, et al. Berotralstat Positively Impacts Patient-Reported Satisfaction: Results from the Phase 3 APeX-2 trial; Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology; Poster 158, Nov (2020)