Orladeyo is the first once-daily oral therapy specifically designed to prevent attacks of HAE patients, by decreasing the activity of plasma kallikrein
Orladeyo (berotralstat) capsules 150 mg. (Credit: BioCryst Pharmaceuticals.)
US-based BioCryst Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for Orladeyo (berotralstat) to prevent attacks related to hereditary angioedema (HAE) in patients aged 12 years and above.
Orladeyo is the first orally administered non-steroidal therapy specifically designed to prevent attacks of HAE, by decreasing the activity of plasma kallikrein.
APeX-2, a long-term open-label Phase 3 trial, demonstrated that Orladeyo has significantly reduced the attacks in HAE patients at 24 weeks, and the reduction was sustained with a mean attack rate of 0.8 attacks per month, at 48 weeks.
APeX-2 trial investigator Marc Riedl said: “Patients and physicians acknowledge that HAE treatments can add a burden to patients’ lives. As an oral, once-daily option, ORLADEYO can provide significant attack reduction and lessen the burden associated with injections and infusions.
“With this new treatment option, physicians and patients can continue to have collaborative discussions to choose the treatment that meets each patient’s needs, life circumstances and preferences.”
BioCryst stated that the treatment was well-tolerated and safe in the clinical trial. Gastrointestinal reactions are the most frequently reported adverse reaction compared to placebo.
Apart from the reduction in HAE attack rate, data from APeX-2 also shows that the treatment using Orladeyo resulted in meaningful improvements in both quality of life and overall patient-reported satisfaction in study participants.
Also, the once-daily orladeyo (150 mg) treatment has significantly reduced the monthly use of the standard of care on-demand medicine, said the company.
BioCryst president and chief executive officer Jon Stonehouse said: “The FDA approval of ORLADEYO fulfils a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks.
“Thank you to the HAE patients who participated in our clinical trials, to the investigators around the world who conducted these trials, to the HAEA for their patient advocacy and to our employees who never forgot that patients were waiting.
“We will stay focused on enabling access and providing personalized support to HAE patients and physicians.”