E9-25135. Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications; Availability

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA) is announcing the availability of a guidance document entitled “Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications,” dated October 2009. In this guidance, we refer to these products for hematopoietic reconstitution for specified indications as hematopoietic progenitor cells, cord (HPC-C). This guidance (HPC-C licensure guidance) provides recommendations to manufacturers applying for licensure of minimally manipulated, unrelated allogeneic placental/umbilical cord blood, for specified indications. Elsewhere in this issue of the Federal Register, FDA is publishing a draft guidance entitled “Guidance for Industry and FDA Staff: Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical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.” FDA is also announcing the end of the phased-in implementation period for IND and biologics license application (BLA) requirements for minimally manipulated unrelated allogeneic hematopoietic stem/progenitor cell products. The HPC-C licensure guidance announced in this notice finalizes the draft guidance entitled “Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution in Patients with Hematological Malignancies,” dated December 2006.

DATES:

Submit electronic or written comments on agency guidances at any time. FDA no longer intends to exercise enforcement discretion with respect to IND and BLA requirements for minimally manipulated, unrelated allogeneic hematopoietic stem/progenitor cell products and the phase in implementation period for IND and BLA requirements will end after October 20, 2011.

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 guidance document entitled “Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications,” dated October 2009. This guidance provides recommendations to manufacturers applying for licensure of minimally manipulated, unrelated allogeneic placental/umbilical cord blood, for specified indications. Elsewhere in this issue of the Federal Register, FDA is publishing an IND draft 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.” FDA is also announcing that it no longer intends to exercise enforcement discretion with respect to IND and BLA requirements for minimally manipulated unrelated allogeneic hematopoietic stem/progenitor cell products and the phase-in implementation period for IND and license application requirements for these products will end (see DATES for ending date). We encourage sponsors to send in applications as soon as possible to allow sufficient time for review, comment, and re-submission as needed to complete all actions by the end of this 2-year period.

In the Federal Register notice of January 20, 1998 (63 FR 2985), FDA requested submission of comments proposing establishment controls, process controls, and product standards designed to ensure the safety and effectiveness of minimally manipulated unrelated allogeneic hematopoietic stem/progenitor cell products derived from peripheral and cord blood for hematopoietic reconstitution. Also, in the January 20, 1998, notice, FDA announced its intention to phase in implementation of IND and license application requirements for minimally manipulated unrelated allogeneic hematopoietic stem/progenitor cell products to permit the development of licensing standards for those products where possible.

In the Federal Register notice of January 17, 2007 (72 FR 1999), FDA announced the availability of the draft guidance entitled “Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution in Patients with Hematological Malignancies,” dated December 2006. FDA received comments on the December 2006 draft guidance and those comments were considered as the guidance was finalized. The HPC-C licensure guidance announced in this notice finalizes the December 2006 draft guidance. Some of the comments received by FDA expressed the importance of access and availability of HPC-C products that not do meet standards for licensure and therefore cannot be licensed. FDA recognizes the importance of these products and is publishing a draft IND guidance addressing IND submissions for such products.

This guidance is consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents FDA's current thinking on these topics. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. Alternative approaches may be used if such approaches satisfy 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 in 21 CFR part 201 have been approved under OMB Control No. 0910-0572; 21 CFR part 211 have been approved under OMB Control No. 0910-0139; 21 CFR part 600 have been approved under OMB Control No. 0910-0308; 21 CFR parts 601, 610, and FDA Form 356(h) have been approved under OMB Control No. 0910-0338; 21 CFR part 1271 have been approved under OMB Control Nos. 0910-0559, 0910-0469, and 0910-0543; and FDA Form 3500A has been approved under OMB Control No. 0910-0291.

III. Comments

Interested persons may, at any time, submit to the Division of Dockets Management (see ADDRESSES) electronic or written 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.

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: October 14, 2009.

David Horowitz,

Assistant Commissioner for Policy.