Major Amendment Extension Memo - Ruconest


MEMORANDUM

From:     Elena Karnaukhova, Ph.D.; HFM-343; LBVB, DH, 
           OBRR, CBER; (301) 402-4635, FAX (301) 402-2780
Subject:  Major Amendment Extension Memo for ----(b)(4)----           
for C1 Esterase Inhibitor (Recombinant), rC1INH,  [Rhuconest] submitted by Pharming Group NV 
Through:  Abdu Alayash, Ph.D.; HFM-343, LBVB, DH, OBRR,
           CBER; (301) 827-3813, FAX (301) 435-4034
 Basil Golding, MD, Director; HFM-300; DH, OBRR,
 CBER; (301) 496-4396; FAX (301) 827-3535          
To:       Nannette Cagungun, RPM; HFM-380; RPMB, DBA, OBRR, 
      CBER; (301) 827-6174          
           To the file: STN 125495/0           

Action recommended: 

Extend the review clock for BLA 125495/0 for three months on the basis of major clinical amendment BL ----(b)(4)---- received by CBER on February 6, 2014.

Rationale:
•On February 6, 2014 CBER received Amendment 24 (BL 125462/0/24) with regards to substantive clinical issues communicated to the applicant in the Late-Cycle Meeting package on January 3, 2014.
•This BLA amendment (BL 125462/0/24) to an original application provides for an important efficacy related analysis in response to substantive clinical issues identified by clinical branch during the BLA review.
•On the basis of a substantial amount of the new information submitted to FDA at this time of the review cycle (ADD: April 16, 2014) and in accordance with SOPP 8402, this submission qualifies as a major amendment.
•As the review committee chair, I recommend to extend the review clock for BLA 125495/0 for three months on the basis of major clinical amendment BL ----(b)(4)--- received by CBER on February 6, 2014.
•In addition, on February 14, 2014 CBER received Amendment 25 (BL 125462/0/25) which includes the Validation report for neutralizing antibody assay to address the CMC issue communicated to the sponsor in the Late-Cycle Meeting package on January 3, 2014. The new CMC data provided in BL 125462/0/25 will be reviewed within the extended review time.
