FDA approves innovative oral therapy to prevent hereditary angioedema

. BioCryst Pharmaceuticals announced Thursday that the U.S. FDA has approved Orladeyo as a daily oral therapy to prevent hereditary angioedema (HAE) episodes in adults and pediatric patients over 12 years of age. According to the press release, this is the first oral nonsteroidal treatment option to prevent HAE seizures.Hereditary angioedema is a rare genetic disease, mainly manifested in recurrent swelling of skin, respiratory tract and internal organs, when edema occurs in the airways, can cause laryngeal edema, if not rescued in time, can suffocate to death. When edema occurs in the gastrointestinal tract, it will be similar to the performance of acute abdominal disease, often misdiagnosed as appendicitis, acute pancreatitis, etc., leading to emergency caesarean section. The disease not only has the potential to be life-threatening, but also a heavy financial and spiritual burden on patients and their families.Orladeyo is an innovative oral-specific plasma peptide release enzyme inhibitor. In the critical Phase 3 clinical trial APeX-2, Orladeyo significantly reduced the onset of the disease at 24 weeks of treatment, a decrease that lasted until 48 weeks. The rate of HAE seizures in patients who completed 48 weeks of treatment decreased from an average of 2.9 times per month at baseline to an average of 1.0 per month after 48 weeks of treatment. In the long-term open label APeX-S trial, the average rate of seizures in patients who completed 48 weeks of treatment was 0.8 per month.“ FDA approved Orladeyo to fulfill BioCryst's commitment to HAE patients to help them develop an oral drug that will fulfill their dream of preventing and reducing the burden of disease seizures. Mr. Jon Stonehouse, President and CEO of BioCryst. (Drug Mingkang):. . . BioCryst Announces FDA Approval of ORLADEYO™ (Berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioed Patientemas. Retrieved December 4, 2020, from