Document ID: FDA-2009-D-0490-0016
Agency: fda
Document Type: Notice
Title: Guidance for Industry and Food and Drug Administration Staff:
Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients With Disorders Affecting the Hematopoietic System; Availability
Posted Date: 2014-03-05T05:00Z

[Federal Register Volume 79, Number 43 (Wednesday, March 5, 2014)]
[Notices]
[Pages 12509-12510]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-04812]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2009-D-0490]

Guidance for Industry and Food and Drug Administration Staff: 
Investigational New Drug Applications for Minimally Manipulated, 
Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for 
Hematopoietic and Immunologic Reconstitution in Patients With Disorders 
Affecting the Hematopoietic System; 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 and FDA 
Staff: Investigational New Drug Applications for Minimally Manipulated, 
Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for 
Hematopoietic and Immunologic Reconstitution in Patients With Disorders 
Affecting the Hematopoietic System'' dated March

[[Page 12510]]

2014. The guidance document provides advice to potential sponsors, such 
as cord blood banks, registries, transplant centers, or individual 
physicians serving as sponsor-investigators, to assist in the 
submission of an investigational new drug application (IND) for certain 
hematopoietic progenitor cells from placental/umbilical cord blood 
(HPC, Cord Blood), when such HPC, Cord Blood units are not licensed, 
and when a suitable human leukocyte antigen (HLA) matched cord blood 
transplant is needed for hematopoietic and immunologic reconstitution 
in patients with disorders affecting the hematopoietic system that are 
inherited, acquired, or result from myeloablative treatment and there 
is no satisfactory alternative treatment available. If unlicensed HPC, 
Cord Blood units are made available for clinical use, they must be 
distributed under an IND. The guidance announced in this document 
finalizes the draft guidance of the same title dated June 2013 and 
supersedes the final guidance entitled ``Guidance for Industry and FDA 
Staff: Investigational New Drug Applications (INDs) for Minimally 
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood 
Intended for Hematopoietic Reconstitution for Specified Indications'' 
dated June 2011.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of the guidance to 
the Office of Communication, Outreach and Development (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.
    Submit electronic comments on the guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Tami Belouin, 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 and FDA Staff: Investigational New Drug 
Applications for Minimally Manipulated, Unrelated Allogeneic Placental/
Umbilical Cord Blood Intended for Hematopoietic and Immunologic 
Reconstitution in Patients With Disorders Affecting the Hematopoietic 
System,'' dated March 2014. The guidance provides advice to potential 
sponsors to assist in the submission of an IND for certain HPC, Cord 
Blood, when such HPC, Cord Blood units are not licensed in accordance 
with title 21 of the Code of Federal Regulations part 601 (21 CFR part 
601), and when a suitable HLA matched cord blood transplant is needed 
for hematopoietic and immunologic reconstitution in patients with 
disorders affecting the hematopoietic system that are inherited, 
acquired, or result from myeloablative treatment and there is no 
satisfactory alternative treatment available. If unlicensed HPC, Cord 
Blood units are made available for clinical use, they must be 
distributed under an IND meeting the applicable requirements in 21 CFR 
part 312.
    In the Federal Register of June 17, 2013 (78 FR 36194), FDA 
announced the availability of the draft guidance of the same title 
dated June 2013. FDA received no comments on the draft guidance and 
only editorial changes were made to improve clarity. The guidance 
announced in this notice finalizes the draft guidance dated June 2013 
and supersedes the final guidance entitled ``Guidance for Industry and 
FDA Staff: Investigational New Drug Applications (INDs) for Minimally 
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood 
Intended for Hematopoietic Reconstitution for Specified Indications'' 
dated June 2011.
    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.
    Elsewhere in this issue of the Federal Register, we also are 
announcing the availability of another, related guidance entitled 
``Guidance for Industry: Biologics License Applications for Minimally 
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood 
Intended for Hematopoietic and Immunologic Reconstitution in Patients 
With Disorders Affecting the Hematopoietic System.'' That guidance 
supersedes the document entitled ``Guidance for Industry: Minimally 
Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood 
Intended for Hematopoietic Reconstitution for Specified Indications'' 
dated October 2009.

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 in 21 CFR part 312 have been approved under 
OMB control number 0910-0014; 21 CFR part 56 have been approved under 
OMB control number 0910-0130; 21 CFR part 1271 have been approved under 
OMB control number 0910-0543; and Form FDA 1571 has been approved under 
OMB control number 0910-0014.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the guidance at 
either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: February 27, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-04812 Filed 3-4-14; 8:45 am]
BILLING CODE 4160-01-P