BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) has announced that it has received FDA approval for once-daily, oral ORLADEYO™ for prophylaxis for preventing attacks in hereditary angioedema (HAE)in pediatric patients above 12 years and adults.
ORLADEYO first oral treatment to prevent HAE attacks
Anthony Castaldo, CEO and President of the US Hereditary Angioedema Association, said that ORLADEYO would be the first oral non-steroidal treatment alternative to prevent HAE attacks in patients. This represents a massive milestone and welcome step in delivering more treatment alternatives to patients and physicians.
ORLADEYO demonstrated a significant reduction of HAE attacks after 24 weeks in a pivotal Phase 3 APeX-2 trial, with the reduction sustained through 48 weeks. There was a significant reduction in HAE attacks in patients the completed the 48-weeks 150mg treatment from an average of 2.9 attacks per month to an average of 1.0 attacks per month after treatment. Interestingly, the patients who completed the long-term open-label APeX-S study after 48 weeks showed an average HAE attack rate of 0.8 attacks/month.
There were a favorable safety and tolerability profile in both trials, with the most reported adverse events in patients that received ORLADEYO being gastrointestinal reactions. The reactions occurred at the onset of the treatment and were less frequent and self-resolving with time.
ORLADEYO to reduce the treatment burden for HAE patients
Marc Riedl, clinical director and professor of medicine, Hereditary Angioedema Association Centres at University Of California and investigator in the APeX-2 study, said that HAE treatments could burden patients’ lives. Therefore this once-daily, oral ORLADEYO which offers significant HAE attack reduction, is a massive milestone and will lessen the associated burden with infusions and injections. Riedl added that the new treatment alternative would give physicians an option to meet the needs of patients, preferences, and life circumstances.
BioCryst’s CEO and president, Jon Stonehouse, said that ORLADEYO’s approval fulfills the company’s promise to HAE patients of delivering an oral, once-daily treatment for prevention of attacks. Stonehouse said that they would continue enabling access to the treatment to support HAE patients.