|From:||L. Ross Pierce, M.D, HFM-392|
|To:||Basil Golding, M.D., Director, HFM-330|
|Through:||Nisha Jain, M.D., Chief, HFM-392|
|Subject:||BPAC Waiver for CSL Behring’s BLA For C1-esterase Inhibitor (Plasma- Derived)[Berinert], STN 125287/0|
OBRR reviewed information from this application and determined that referral to the Blood Products Advisory Committee (BPAC) prior to licensure is not needed for the following reasons (FDAAA [HR 3580-138 SEC. 918: REFERRAL TO ADVISORY COMMITTEE]):
- New molecular entity (NME) provision does not apply to Berinert as another plasma-derived C1 esterase inhibitor product was recently presented at BPAC on May 2, 2008 for prophylaxis of acute attacks of hereditary angioedema.
- C1-esterase inhibitor’s mechanism of action and function in the blood complement, fibrinolytic, and coagulation cascades are well studied and understood.
- Plasma-derived C1-esterase inhibitor concentrates have been used to treat HAE attacks for over 25 years in countries where these products are available.
- Berinert is C1-esterase inhibitor product derived from US Source Plasma that has been approved for many years in Europe.
- The study design to evaluate efficacy of Berinert was adequate and the results of the study did not raise any concerns related to safety. Berinert has demonstrated a favorable safety profile.
- Review of information submitted in the BLA for Berinert indicated efficacy for the overall study population of subjects with abdominal and facial attacks, as well as the subgroup of subjects with abdominal attacks. BPAC discussion of this application is unlikely to change the outcome of the review of this file from a regulatory standpoint.
- Berinert is the first C1 inhibitor product approved for acute treatment of HAE attacks and, therefore, it is in the interest of the public health not to delay licensure by requiring consideration by BPAC of this application.
Felice D’Agnillo, Ph.D., HFM-343