2014-04812. 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 ...

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 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 The guidance announced in this document finalizes the draft guidance of the same title dated June 2013 and supersedes the final guidance entitled “Guidance for Industry and FDA Staff: Investigational New Drug Applications (INDs)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.