Document ID: FDA-2005-D-0208-0003
Agency: fda
Document Type: Notice
Title: Guidance for Industry: Safety, Efficacy, and Pharmacokinetic Studies to Support Marketing of Immune Globulin Intravenous (Human) as Replacement Therapy for Primary Humoral Immunodeficiency; Availability
Posted Date: 2008-07-17T04:00Z

[Federal Register: July 17, 2008 (Volume 73, Number 138)]
[Notices]               
[Page 41087-41088]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr17jy08-83]                         

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

 
[Docket No. FDA-2005-D-0208] (formerly Docket No. 2005D-0438)

Guidance for Industry: Safety, Efficacy, and Pharmacokinetic 
Studies to Support Marketing of Immune Globulin Intravenous (Human) 
as Replacement Therapy for Primary Humoral Immunodeficiency; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY:  The Food and Drug Administration (FDA) is announcing the 
availability of a document entitled ``Guidance for Industry: Safety, 
Efficacy, and Pharmacokinetic Studies to Support Marketing of Immune 
Globulin Intravenous (Human) as Replacement Therapy for Primary Humoral 
Immunodeficiency,'' dated June 2008. The guidance document provides 
recommendations for the design of clinical trials to assess the safety, 
efficacy, and pharmacokinetics of immune globulin intravenous (human) 
(IGIV) products as replacement therapy in primary humoral 
immunodeficiency. The guidance announced in this notice finalizes the 
draft guidance of the same title dated November 2005.

DATES:  Submit written or electronic comments on agency guidances at 
any time. Submit written requests for single copies of the guidance to 
the Office of Communication, Training, and Manufacturers Assistance 
(HFM-40), Center for Biologics Evaluation and Research (CBER), Food and 
Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 
20852-1448. Send one self-addressed adhesive label to assist the office 
in processing your requests. The guidance may also be obtained by mail 
by calling CBER at 1-800-835-4709 or 301-827-1800. See the 
SUPPLEMENTARY INFORMATION section for electronic access to the guidance 
document.

ADDRESSES: Submit written comments on the guidance to the Division of 
Dockets Management (HFA-305), Food and Drug Administration, 5630 
Fishers Lane, rm. 1061, Rockville, MD 20852. Submit electronic comments 
to http://www.regulations.gov.

FOR FURTHER INFORMATION CONTACT: Denise S[aacute]nchez, Center for 
Biologics Evaluation and Research (HFM-17), Food and Drug 
Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-
1448, 301-827-6210.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a document entitled 
``Guidance for Industry: Safety, Efficacy, and Pharmacokinetic Studies 
to Support Marketing of Immune Globulin Intravenous (Human) as 
Replacement Therapy for Primary Humoral Immunodeficiency,'' dated June 
2008. This guidance provides investigational new drug application (IND) 
and biologics license application (BLA) sponsors with recommendations 
for the design of clinical trials to assess the safety, efficacy, and 
pharmacokinetics of investigational IGIV products when used as 
replacement therapy in primary humoral immunodeficiency. This

[[Page 41088]]

guidance is intended to assist sponsors in the preparation of the 
clinical/biostatistical and human pharmacokinetic sections of a BLA. 
This guidance does not address additional sections of a BLA, such as 
chemistry, manufacturing, and controls and pre-clinical toxicology, for 
an IGIV product for this indication.
    In the Federal Register of December 1, 2005 (70 FR 72124), FDA 
announced the availability of the draft guidance of the same title 
dated November 2005. FDA received several comments on the draft 
guidance and those comments were considered as the guidance was 
finalized. A summary of changes includes: Recommendations for 
compliance with the Pediatric Research Equity Act of 2007, refinements 
to the criteria for diagnosing serious infections, refinements to the 
recommended safety analyses of adverse experiences temporally related 
to infusions, and additional guidance on the methodology of 
pharmacokinetic studies. The guidance announced in this notice 
finalizes the draft guidance dated November 2005.
    The guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents FDA's 
current thinking on this topic. It does not create or confer any rights 
for or on any person and does not operate to bind FDA or the public. An 
alternative approach may be used if such approach satisfies the 
requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The 
collections of information regarding BLAs (21 CFR part 601) have been 
approved under OMB control number 0910-0338.

III. Comments

    Interested persons may, at any time, submit to the Division of 
Dockets Management (see ADDRESSES) written or electronic comments 
regarding the guidance. Submit a single copy of electronic comments or 
two paper copies of any mailed comments, except that individuals may 
submit one paper copy. Comments are to be identified with the docket 
number found in brackets in the heading of this document. A copy of the 
guidance and received comments are available for public examination in 
the Division of Dockets Management between 9 a.m. and 4 p.m., Monday 
through Friday.
    Please note that on January 15, 2008, the FDA Division of Dockets 
Management Web site transitioned to the Federal Dockets Management 
System (FDMS). FDMS is a Government-wide, electronic docket management 
system. Electronic comments or submissions will be accepted by FDA only 
through FDMS at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the guidance at 
either http://www.fda.gov/cber/guidelines.htm or http://
www.regulations.gov.

    Dated: July 11, 2008.
Jeffrey Shuren,
Associate Commissioner for Policy and Planning.
[FR Doc. E8-16395 Filed 7-16-08; 8:45 am]

BILLING CODE 4160-01-S