According to Lev, it is the first company to submit a licensing application for the acute treatment of hereditary angioedema (HAE) in the US. The FDA has sixty days to determine whether the BLA is sufficiently complete to be filed for substantive review.
US biopharmaceutical company Lev is developing Cinryze (C1 inhibitor) as a comprehensive replacement therapy for the treatment of HAE, or C1 inhibitor deficiency, a potentially life-threatening genetic disease characterized by recurrent attacks of inflammation. Lev is developing Cinryze both for the treatment of acute HAE attacks and as a prophylactic therapy to prevent HAE attacks.
In March, Lev announced the successful completion of its pivotal phase III clinical trial for the acute treatment of HAE, in which the protocol-defined primary endpoint was achieved, indicating a clinically and statistically significant reduction in the time to relief of acute HAE symptoms. There is currently no FDA-licensed acute therapy for HAE in the US.
Lev reiterated that top-line results from the prophylactic portion of its phase III study are expected to be made available during the second half of this year. If positive and supportive, such data may be submitted as an amendment to the BLA to seek an expanded label indication for Cinryze to include the prevention of HAE attacks.