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, 12509-12510 [2014-04812]

Download as PDF Federal Register / Vol. 79, No. 43 / Wednesday, March 5, 2014 / Notices mstockstill on DSK4VPTVN1PROD with NOTICES applicable regulatory requirements. The guidance announced in this document finalizes the draft guidance of the same title dated June 2013 and supersedes the guidance entitled ‘‘Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic Placental/ Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications’’ dated October 2009. 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 https://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: 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’’ dated March 2014. The guidance document provides recommendations for manufacturers to apply for licensure of minimally manipulated, unrelated allogeneic placental/umbilical cord blood, for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. The guidance document is intended to assist manufacturers obtain a biologics license. The guidance contains VerDate Mar<15>2010 17:13 Mar 04, 2014 Jkt 232001 information about the manufacture of minimally manipulated, unrelated, allogeneic placental/umbilical cord blood and how to comply with applicable regulatory requirements. In the Federal Register of June 17, 2013 (78 FR 36196), FDA announced the availability of the draft guidance of the same title dated June 2013. FDA received a few comments on the draft guidance and those comments were considered as the guidance was finalized. Minor changes were made to improve clarity. The guidance announced in this notice finalizes the draft guidance of the same title dated June 2013 and supersedes the guidance entitled ‘‘Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications’’ dated October 2009. 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 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.’’ That guidance supersedes the document 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. 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 201 have been approved under OMB control number 0910–0572; 21 CFR part 211 have been approved under OMB control number 0910–0139; 21 CFR part 600 have been approved PO 00000 Frm 00048 Fmt 4703 Sfmt 4703 12509 under OMB control number 0910–0308; 21 CFR parts 601 and 610, and Form FDA 356h have been approved under OMB control number 0910–0338; and 21 CFR part 1271 have been approved under OMB control number 0910–0543. III. Comments Interested persons may submit either electronic comments regarding this document to https://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 https:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the guidance at either https://www.fda.gov/Biologics BloodVaccines/GuidanceCompliance RegulatoryInformation/Guidances/ default.htm or https:// www.regulations.gov. Dated: February 27, 2014. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2014–04813 Filed 3–4–14; 8:45 am] BILLING CODE 4160–01–P 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. 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 SUMMARY: E:\FR\FM\05MRN1.SGM 05MRN1 mstockstill on DSK4VPTVN1PROD with NOTICES 12510 Federal Register / Vol. 79, No. 43 / Wednesday, March 5, 2014 / Notices 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 https://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: VerDate Mar<15>2010 17:13 Mar 04, 2014 Jkt 232001 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 PO 00000 Frm 00049 Fmt 4703 Sfmt 9990 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 https://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 https:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the guidance at either https://www.fda.gov/Biologics BloodVaccines/GuidanceCompliance RegulatoryInformation/Guidances/ default.htm or https:// 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 E:\FR\FM\05MRN1.SGM 05MRN1

Agencies

[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]

-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 https://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 https://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 https://www.regulations.gov.

IV. Electronic Access

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

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