Document ID: FDA-2015-D-1246-0001
Agency: fda
Document Type: Notice
Title: Investigational Enzyme Replacement Therapy Products: Nonclinical
Assessment; Draft Guidance for Industry; Availability
Posted Date: 2015-05-13T04:00Z

[Federal Register Volume 80, Number 92 (Wednesday, May 13, 2015)]
[Notices]
[Pages 27319-27320]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-11539]

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-1246]

Investigational Enzyme Replacement Therapy Products: Nonclinical 
Assessment; Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled 
``Investigational Enzyme Replacement Therapy Products: Nonclinical 
Assessment.'' This draft guidance is intended to advise the sponsors 
and individuals involved in the design and implementation of 
nonclinical studies on the substance and scope of nonclinical 
information needed to support first-in-human clinical trials, ongoing 
clinical development, and eventual approval of enzyme replacement 
therapy (ERT) products for the treatment of rare, life-threatening 
conditions.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by July 13, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993-0002. Send one self-addressed adhesive label to assist that 
office in processing your requests. See the SUPPLEMENTARY INFORMATION 
section for electronic access to the draft guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov.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: Sushanta Chakder, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 5108, Silver Spring, MD 20993-0002, 301-
796-0861.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Investigational Enzyme Replacement Therapy Products: 
Nonclinical Assessment.''
    This draft guidance provides sponsors and individuals involved in 
the design and implementation of nonclinical studies with 
recommendations on the nonclinical information needed to support 
initiation of clinical trials, ongoing clinical development, and 
eventual licensure or approval for investigational ERT products. The 
recommendations in this guidance are applicable to ERT products 
indicated for lysosomal storage diseases or other diseases related to 
inborn errors of metabolism.
    Because of the wide array of clinical indications, natural history 
of disease, and product types, no single nonclinical program can be 
designed to address all ERT products, and a case-by-case approach to 
both toxicological evaluation and clinical development is warranted to 
optimize and expedite drug development. Common nonclinical issues, such 
as the number of animal species needed for safety assessment, selection 
of animal models and duration of the toxicology studies needed to 
support first-in-human trials, and nonclinical study requirements for 
ultimate licensure or market approval of the ERT product, are addressed 
in this guidance.
    This guidance is intended as an adjunct to the ICH guidances for 
industry entitled ``M3(R2) Nonclinical Safety Studies for the Conduct 
of Human Clinical Trials and Marketing Authorization for 
Pharmaceuticals,'' ``M3(R2) Nonclinical Safety Studies for the Conduct 
of Human Clinical Trials and Marketing Authorization for 
Pharmaceuticals--Questions and Answers,'' and ``S6 Preclinical Safety 
Evaluation of Biotechnology-Derived Pharmaceuticals.''
    This draft guidance is being issued consistent with FDA's good 
guidance

[[Page 27320]]

practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the current thinking of FDA on nonclinical 
assessment of investigational ERT products. It does not establish any 
rights for any person and is not binding on FDA or the public. You can 
use an alternative approach if it satisfies the requirements of the 
applicable statutes and regulations.

II. The Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information that 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, and the information 
collection in the regulations on good laboratory practice for 
nonclinical laboratory studies (21 CFR part 58) is approved under OMB 
control number 0910-0119.

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 document at 
either http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: May 7, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-11539 Filed 5-12-15; 8:45 am]
 BILLING CODE 4164-01-P