The Ministry of Health, Labour and Welfare (MHLW) in Japan has granted manufacturing and marketing authorization for Firazyr for the acute treatment of HAE attacks in adult patients.
“As a long-term partner to the HAE community, we continually strive to bring treatments to those living with HAE around the world,” said Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire plc. “HAE attacks can be unpredictable and debilitating and we are delighted that, subject to price listing, we will be able to provide the Japanese HAE community with the first subcutaneous on-demand therapy to treat acute HAE attacks.”
The use of Firazyr in Japanese patients was examined in an open-label, single-arm, Phase 3 study of 8 adult patients with a confirmed diagnosis of HAE who experienced angioedema attacks. During the study, 3 patients self-administered Firazyr and 5 patients had Firazyr administered by a physician. The primary efficacy endpoint was time to onset of symptom relief (TOSR), defined as a 50% reduction from baseline in patient Visual Analog Scale (VAS) score.
The study showed that Firazyr was well tolerated and demonstrated symptom relief during an acute HAE attack through a single injection. Overall, median TOSR was 1.75 hours, and TOSR was similar for patients who self-administered Firazyr or who had Firazyr administered by a physician. Symptom relief was attained as early as 1 hour after Firazyr injection and all patients had symptom relief within 5 hours.
The most common adverse events in patients treated with Firazyr were injection site reactions such as erythema, or swelling, which were found to be mild to moderate in severity.