Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment; Draft Guidance for Industry; Availability, 27319-27320 [2015-11539]

Download as PDF Federal Register / Vol. 80, No. 92 / Wednesday, May 13, 2015 / Notices III. Requests for Comments A. General Information About Submitting 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. IV. Electronic Access Persons with access to the Internet may obtain an electronic version of the draft guidance at either https:// www.regulations.gov or https:// www.fda.gov/TobaccoProducts/ GuidanceComplianceRegulatory Information/default.htm. Dated: May 8, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–11538 Filed 5–12–15; 8:45 am] BILLING CODE 4164–01–P B. Public Availability of Comments 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. As a matter of Agency practice, FDA generally does not post comments submitted by individuals in their individual capacity on https://www.regulations.gov. This is determined by information indicating that the submission is written by an individual, for example, the comment is identified with the category ‘‘Individual Consumer’’ under the field titled ‘‘Category (Required),’’ on the ‘‘Your Information’’ page on https:// www.regulations.gov. For this docket, however, FDA will not be following this general practice. Instead, FDA will post on https://www.regulations.gov comments to this docket that have been submitted by individuals in their individual capacity. If you wish to submit any information under a claim of confidentiality, please refer to 21 CFR 10.20. asabaliauskas on DSK5VPTVN1PROD with NOTICES C. Information Identifying the Person Submitting the Comment Please note that your name, contact information, and other information identifying you will be posted on https:// www.regulations.gov if you include that information in the body of your comments. For electronic comments submitted to https:// www.regulations.gov, FDA will post the body of your comment on https:// www.regulations.gov along with your state/province and country (if provided), the name of your representative (if any), and the category identifying you (e.g., individual, consumer, academic, industry). For written submissions submitted to the Division of Dockets Management, FDA will post the body of your comments on https://www.regulations.gov, but you can put your name and/or contact information on a separate cover sheet and not in the body of your comments. VerDate Sep<11>2014 17:27 May 12, 2015 Jkt 235001 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. 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. SUMMARY: PO 00000 Frm 00036 Fmt 4703 Sfmt 4703 27319 Submit electronic comments on the draft 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: 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 E:\FR\FM\13MYN1.SGM 13MYN1 27320 Federal Register / Vol. 80, No. 92 / Wednesday, May 13, 2015 / Notices 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. Dated: May 7, 2015. Leslie Kux, Associate Commissioner for Policy. II. The Paperwork Reduction Act of 1995 [Docket No. FDA–2015–N–1339] 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 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 document at either https://www.fda.gov/Drugs/Guidance ComplianceRegulatoryInformation/ Guidances/default.htm or https:// www.regulations.gov. [FR Doc. 2015–11539 Filed 5–12–15; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration Determination That SODIUM SULAMYD (sulfacetamide sodium) Ophthalmic Solution and Ophthalmic Ointment Were Not Withdrawn From Sale for Reasons of Safety or Effectiveness AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA or Agency) has determined that the drug products listed in this document were not withdrawn from sale for reasons of safety or effectiveness. This determination means that FDA will not begin procedures to withdraw approval of abbreviated new drug applications (ANDAs) that refer to these drug products, and it will allow FDA to continue to approve ANDAs that refer to the products as long as they meet relevant legal and regulatory requirements. FOR FURTHER INFORMATION CONTACT: Amy Hopkins, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6223, Silver Spring, MD 20993–0002, 301– 796–5418, Amy.Hopkins@fda.hhs.gov. SUPPLEMENTARY INFORMATION: In 1984, Congress enacted the Drug Price Competition and Patent Term Restoration Act of 1984 (Pub. L. 98–417) (the 1984 amendments), which authorized the approval of duplicate versions of drug products approved under an ANDA procedure. ANDA applicants must, with certain exceptions, show that the drug for which they are seeking approval SUMMARY: contains the same active ingredient in the same strength and dosage form as the ‘‘listed drug,’’ which is a version of the drug that was previously approved. ANDA applicants do not have to repeat the extensive clinical testing otherwise necessary to gain approval of a new drug application (NDA). The 1984 amendments include what is now section 505(j)(7) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(j)(7)), which requires FDA to publish a list of all approved drugs. FDA publishes this list as part of the ‘‘Approved Drug Products with Therapeutic Equivalence Evaluations,’’ which is generally known as the ‘‘Orange Book.’’ Under FDA regulations, a drug is removed from the list if the Agency withdraws or suspends approval of the drug’s NDA or ANDA for reasons of safety or effectiveness or if FDA determines that the listed drug was withdrawn from sale for reasons of safety or effectiveness (21 CFR 314.162). Under § 314.161(a) (21 CFR 314.161(a)), the Agency must determine whether a listed drug was withdrawn from sale for reasons of safety or effectiveness: (1) Before an ANDA that refers to that listed drug may be approved, (2) whenever a listed drug is voluntarily withdrawn from sale and ANDAs that refer to the listed drug have been approved, and (3) when a person petitions for such a determination under 21 CFR 10.25(a) and 10.30. Section 314.161(d) provides that if FDA determines that a listed drug was withdrawn from sale for safety or effectiveness reasons, the Agency will initiate proceedings that could result in the withdrawal of approval of the ANDAs that refer to the listed drug. FDA has become aware that the drug products listed in the table in this document are no longer being marketed. (As requested by the applicant, FDA withdrew approval of NDA 005963 for SODIUM SULAMYD (sulfacetamide sodium) Ophthalmic Solution and Ophthalmic Ointment in the Federal Register of August 16, 2001 (66 FR 43017)). Drug Applicant NDA 005963 ........................ asabaliauskas on DSK5VPTVN1PROD with NOTICES Application No. SODIUM SULAMYD (sulfacetamide sodium) Ophthalmic Solution 10%, Ophthalmic Solution 30%, and Ophthalmic Ointment 10%. Schering Plough Corp., 2000 Galloping Hill Rd., Kenilworth, NJ 07033. FDA has reviewed its records and, under § 314.161, has determined that the drug products listed in this document were not withdrawn from sale for reasons of safety or VerDate Sep<11>2014 17:27 May 12, 2015 Jkt 235001 effectiveness. Accordingly, the Agency will continue to list the drug products listed in this document in the ‘‘Discontinued Drug Product List’’ section of the Orange Book. The PO 00000 Frm 00037 Fmt 4703 Sfmt 4703 ‘‘Discontinued Drug Product List’’ identifies, among other items, drug products that have been discontinued from marketing for reasons other than safety or effectiveness. E:\FR\FM\13MYN1.SGM 13MYN1

Agencies

[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 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: 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 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 document at 
either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or https://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
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