The European Commission (EC) has approved oral, once-daily Orladeyo (berotralstat) for the prevention of recurrent HAE attacks in 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,” says 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,” says Henrik Balle Boysen, Executive Vice President and COO of HAE International.
The EC approval of Orladeyo is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.
BioCryst Pharmaceuticals, Inc. 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,” says Jon Stonehouse, President and CEO 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.”
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).