From President Beverley Yamamoto, HAE Japan (HAEJ):
HAEJ applied for the 3rd PASE Award from EFPIA (European Federation of Pharmaceutical Industries and Associations) – PASE is Patient Advocacy Support by EFPIA. HAEJ applied for a project on paediatric HAE to do a survey to identify patients and their treatment needs as well as to carry out focus groups with families and school representatives in order to identify the kinds of resources needed to support children in schools.
HAEJ received one of two grand awards: http://www.efpia.jp/link/190902_PASE_ResultAnnouncement_ENG_Final.pdf
HAEJ received the grand award for the 2nd PASE awards in 2018, and on 13 November Makiko Matusyama and I gave a presentation of the results of the research on intravenous self-injection training to the EFPIA Board and guests to the event, which included a number of patient advocacy organizations. We produced a report in English on the project as well as a Japanese translation: “Stakeholder expectations and concerns regarding intravenous self-infusion training for injection of C1 Esterase Inhibitor (C1-inh) for patients with Hereditary Angioedema”.
The national financial daily newspaper, The Nikkei Shinbun, ran a series of four articles in October and November 2019 focusing on HAE and the activities of HAEJ.
In a press release on 5 November 2019, Biocryst announced its plans to submit a Japanese New Drug application (JNDA) for BCX7353 to the Pharmaceuticals and Medical Devices Agency (PMDA) in the first quarter of 2020. Biocryst are partnering with Torrii Pharmaceuticals to commercialize BCX7353 in Japan.
If all goes to plan, patients in Japan will have access to a long-term prophylactic treatment for HAE for the first time.