Hereditary angioedema (HAE) the first oral therapy! FDA approves plasma kinetic peptide release enzyme inhibitor Orladeyo (berotralstat)!

HAE Seizures (Photo Source: genassistabcs.com) December 05, 2020 // -- BioCryst Pharmaceuticals is working to discover new, oral, and small molecule drugs for treating rare diseases in which enzymes play a key role in the biological pathf path of disease and have significantly unfolested medical needs.
recently, the company announced that the U.S. Food and Drug Administration (FDA) has approved Orladeyo capsule, a daily oral drug used in children and adults as young as ≥12 years of age, to prevent hereditary angioedema (HAE) seizures.
it's worth noting that Orladeyo is the first targeted oral therapy approved to prevent HAE seizures, and its approval will make significant progress in the treatment of HAE patients and help reduce the burden of treatment for patients.
orladeyo is also under review by EU and Japanese regulators.
Orladeyo's active pharmaceutical ingredient is belotralstat, a new, oral, daily, powerful, selective human plasma peptide release enzyme inhibitor that is in late clinical development for HAE patients to prevent and treat angioedema attacks.
, BioCryst is currently developing two preparations for berotralstat, capsules for the prevention of HAE seizures, and oral liquid preparations are currently being developed clinically in Phase II for the treatment of acute HAE seizures.
in the key Phase 3 APeX-2 trial, Orladeyo significantly reduced HAE seizures during the 24th week of treatment, and this decrease continued into week 48.
HAE patients who completed 48 weeks of treatment (150 mg) had an average of 2.9 seizures per month at baseline check-ups and reduced to an average of 1.0 seizures per month after 48 weeks of treatment.
patients who completed 48 weeks of treatment (150 mg) in a long-term open-label APeX-S trial had an average of 0.8 seizures per month.
in 2 trials, Orladeyo was safe and well-to-bear.
the most commonly reported adverse reactions were gastrointestinal reactions in patients who took Oral Orladeyo compared to the placebo group.
these reactions usually occur early after Orladeyo's treatment begins, become less frequent over time, and usually disappear on their own.
there is a significant therapeutic burden with existing HAE prophylac treatments.
In addition to reducing HAE seizure rates, data from the APeX-2 trial showed significant improvements in quality of life and overall patient satisfaction in patients who took oral Orladeyo (150 mg) once a day, and a significant reduction in the use of standard care-on-demand medications per month.
"Patients and doctors agree that HAE treatment increases the burden on patients," said Marc Riedl, a medical professor and clinical director at the Center for Genetic Angioedema at the University of California, San Diego, and an investigator on the APeX-2 trial.
as a daily oral treatment, Orladeyo can significantly reduce seizures and reduce the burden associated with injections and infusions.
"The approval of Orladeyo will provide HAE patients and their doctors with the first oral nonsteroidal drug to prevent HAE seizures, an important and welcome step in enabling doctors and patients to have more treatment options," said Anthony J. Castaldo, President and CEO of haEA.
() Original Origin: BioCryst Announces FDA approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Anedema Patients