[Federal Register Volume 79, Number 43 (Wednesday, March 5, 2014)]
[Notices]
[Pages 12509-12510]
From the Federal Register Online via the Government Publishing 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finalizes the draft guidance of the same title dated June 2013 and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]
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