TM for prophylactic treatment of HAE?
The Committee unanimously agreed that the safety and efficacy evidence is sufficient for approval of CinryzeTM for prophylactic treatment of HAE. 2. If the answer to Question #1 is yes, should post-marketing studies be performed to further evaluate the following: a) the optimal dose for prophylaxis in males and females b) immunogenicity c) long-term safety The Committee commented that a registry for HAE may be valuable for long term surveillance of the patient population, including health outcomes, social parameters, cost and reimbursement of product. Members commented that a Congressional mandate established surveillance in the Hemophilia community and an active surveillance system could benefit the HAE patient community. One committee member encouraged FDA to work with the sponsor to examine the existing data to determine to what extent severe attacks were prevented. Additionally, it was recommended that post-marketing studies should be conducted to determine long-term safety (especi
