May 28, 2024

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Plasma kallikrein inhibitor Orladeyo (berotralstat) was approved by EU!

Plasma kallikrein inhibitor Orladeyo (berotralstat) was approved by EU! The first oral treatment for hereditary angioedema (HAE)! 

Plasma kallikrein inhibitor Orladeyo (berotralstat) was approved by EU!   BioCryst Pharmaceuticals announced the key phase 3 APeX-2 trial of Orladeyo (berotralstat, capsule) for the treatment of hereditary angioedema (HAE) at the 2021 European Society of Allergy and Clinical Immunology (EAACI) online-offline mixed conference. 

The results showed that patients who were randomly assigned to receive a 150 mg oral dose of Orladeyo once a day at the beginning of the trial had an average monthly attack rate of 80% lower than baseline during Weeks 25-96.

During the same period, 16 out of 17 months, the median number of seizures also decreased from 2.7 per month at baseline to 0.0 per month.



Compared with part 1 (0-24 weeks) and part 2 (25-48 weeks) of the trial, Orladeyo was well tolerated and reported drug-related adverse events during treatment in part 3 (49-96 weeks) less. Of the patients who entered the trial, 81% completed the trial.

William Sheridan, Chief Medical Officer of BioCryst, said: “The long-term data from our clinical program for 2 years has strengthened Orladeyo’s significant and sustained long-term efficacy and safety in reducing HAE episodes. These results are in line with the fact that many patients in the real world have seen Orladeyo since we launched Orladeyo. The experience is consistent. Orladeyo is a key driver in addressing the needs of patients. We have seen patients shift from injectable preventive drugs and injectable acute treatment drugs to oral Orladeyo once a day to control HAE attacks.”


Orladeyo is the first targeted oral therapy approved for the prevention of HAE attacks. This medicine is an oral, once-a-day medicine used for children and adults aged 12 years and older to prevent HAE attacks. In December 2020, January 2021, and April 2021, Orladeyo was approved in the United States, Japan, and the European Union respectively. The approval of the drug on the market has brought significant progress to the treatment of HAE patients who have been waiting for oral preventive treatment options. Will help reduce the burden of treatment for patients.

The active pharmaceutical ingredient of Orladeyo is berotralstat, which is a new, oral, once-a-day, potent and selective inhibitor of human plasma kallikrein, which works by reducing the activity of plasma kallikrein. The drug was developed for HAE patients to prevent and treat the onset of angioedema. Currently, BioCryst is developing two formulations of berotralstat, capsules are developed to prevent HAE attacks, and oral liquid formulations are used to treat acute HAE attacks.

Plasma kallikrein inhibitor Orladeyo (berotralstat) was approved by EU!

berotralstat mechanism of action

Reduced on-demand medication for HAE patients treated with Orladeyo (150 mg) in the APeX-2 trial: In patients who received Orladeyo 150 mg orally once a day and whose seizure rate decreased by ≥50% from baseline, on-demand medication was used (Dose/month) decreased by 78% from baseline to week 24. In patients whose seizure rate decreased by ≥70% from baseline, on-demand medication decreased by 85% from baseline to week 24, and monthly on-demand medication decreased by 2.2 doses.

Orladeyo-treated adolescent patients with HAE showed a persistently low attack rate: in the open-label safety study APeX-S, an analysis of adolescent patients (age 12-17 years old) treated with Orladeyo 150 mg orally once a day showed that the fourth The weekly average seizure rate (SEM) is 0.4 times/month, which usually lasts until the 48th week. During the 48 weeks of treatment, the average seizure rate of these adolescent patients was 0.0 attacks per month. More than 70% of patients had no seizures from the 4th to the 48th week. Orladeyo was well tolerated in the APeX-S study.

Orladeyo exhibits a persistently low HAE attack rate during COVID-19: Stress is the trigger for HAE attacks, and recently published doctor and patient survey data show that patients reported HAE attack rates due to increased stress associated with the COVID-19 pandemic Increase (before COVID: 1.5 times/3 months, during COVID: 4.4 times/3 months). In the APeX-S trial, an analysis of HAE patients who received oral Orladeyo 150mg once a day treatment showed that the monthly HAE attack rate, including before COVID and during COVID, always remained at a low level (<1 time/ month). Patients treated with Orladeyo maintain a low seizure rate during periods of high social stress and disturbance.

(source:internet, reference only)

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