80 FR 27319 - Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 92 (May 13, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 92 (May 13, 2015)
| Page Range | 27319-27320 | |
| FR Document | 2015-11539 |
[Federal Register Volume 80, Number 92 (Wednesday, May 13, 2015)] [Notices] [Pages 27319-27320] From the Federal Register Online [www.thefederalregister.org] [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[Docket No. FDA-2015-D-1246] Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment.'' This draft guidance is intended to advise the sponsors and individuals involved in the design and implementation of nonclinical studies on the substance and scope of nonclinical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comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by July 13, 2015. ADDRESSES: Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th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 See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. Submit electronic comments on the draft guidance to http://www.regulations.gov.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
![Federal Register / Vol. 80, No. 92 / Wednesday, May 13, 2015 / Notices 27319
III. Requests for Comments IV. Electronic Access Submit electronic comments on the
Persons with access to the Internet draft guidance to http://
A. General Information About www.regulations.gov. Submit written
Submitting Comments may obtain an electronic version of the
draft guidance at either http:// comments to the Division of Dockets
Interested persons may submit either www.regulations.gov or http:// Management (HFA–305), Food and Drug
electronic comments regarding this www.fda.gov/TobaccoProducts/ Administration, 5630 Fishers Lane, Rm.
document to http://www.regulations.gov GuidanceComplianceRegulatory 1061, Rockville, MD 20852.
or written comments to the Division of Information/default.htm. FOR FURTHER INFORMATION CONTACT:
Dockets Management (see ADDRESSES). It Sushanta Chakder, Center for Drug
Dated: May 8, 2015.
is only necessary to send one set of Evaluation and Research, Food and
Leslie Kux,
comments. Identify comments with the Drug Administration, 10903 New
Associate Commissioner for Policy. Hampshire Ave., Bldg. 22, Rm. 5108,
docket number found in brackets in the [FR Doc. 2015–11538 Filed 5–12–15; 8:45 am]
heading of this document. Silver Spring, MD 20993–0002, 301–
BILLING CODE 4164–01–P 796–0861.
B. Public Availability of Comments SUPPLEMENTARY INFORMATION:
Received comments may be seen in DEPARTMENT OF HEALTH AND I. Background
the Division of Dockets Management HUMAN SERVICES FDA is announcing the availability of
between 9 a.m. and 4 p.m., Monday a draft guidance for industry entitled
through Friday, and will be posted to Food and Drug Administration ‘‘Investigational Enzyme Replacement
the docket at http:// [Docket No. FDA–2015–D–1246] Therapy Products: Nonclinical
www.regulations.gov. As a matter of Assessment.’’
Agency practice, FDA generally does Investigational Enzyme Replacement This draft guidance provides sponsors
not post comments submitted by Therapy Products: Nonclinical and individuals involved in the design
individuals in their individual capacity Assessment; Draft Guidance for and implementation of nonclinical
on http://www.regulations.gov. This is Industry; Availability studies with recommendations on the
determined by information indicating AGENCY: Food and Drug Administration, nonclinical information needed to
that the submission is written by an HHS. support initiation of clinical trials,
individual, for example, the comment is ongoing clinical development, and
ACTION: Notice. eventual licensure or approval for
identified with the category ‘‘Individual
Consumer’’ under the field titled SUMMARY: The Food and Drug investigational ERT products. The
‘‘Category (Required),’’ on the ‘‘Your Administration (FDA or Agency) is recommendations in this guidance are
Information’’ page on http:// announcing the availability of a draft applicable to ERT products indicated for
www.regulations.gov. For this docket, guidance for industry entitled lysosomal storage diseases or other
however, FDA will not be following this ‘‘Investigational Enzyme Replacement diseases related to inborn errors of
general practice. Instead, FDA will post Therapy Products: Nonclinical metabolism.
on http://www.regulations.gov Assessment.’’ This draft guidance is Because of the wide array of clinical
comments to this docket that have been intended to advise the sponsors and indications, natural history of disease,
submitted by individuals in their individuals involved in the design and and product types, no single nonclinical
individual capacity. If you wish to implementation of nonclinical studies program can be designed to address all
submit any information under a claim of on the substance and scope of ERT products, and a case-by-case
confidentiality, please refer to 21 CFR nonclinical information needed to approach to both toxicological
10.20. support first-in-human clinical trials, evaluation and clinical development is
ongoing clinical development, and warranted to optimize and expedite
C. Information Identifying the Person eventual approval of enzyme drug development. Common nonclinical
Submitting the Comment replacement therapy (ERT) products for issues, such as the number of animal
the treatment of rare, life-threatening species needed for safety assessment,
Please note that your name, contact selection of animal models and duration
information, and other information conditions.
of the toxicology studies needed to
identifying you will be posted on http:// DATES: Although you can comment on
support first-in-human trials, and
www.regulations.gov if you include that any guidance at any time (see 21 CFR nonclinical study requirements for
information in the body of your 10.115(g)(5)), to ensure that the Agency ultimate licensure or market approval of
comments. For electronic comments considers your comment on this draft the ERT product, are addressed in this
submitted to http:// guidance before it begins work on the guidance.
www.regulations.gov, FDA will post the final version of the guidance, submit This guidance is intended as an
body of your comment on http:// either electronic or written comments adjunct to the ICH guidances for
www.regulations.gov along with your on the draft guidance by July 13, 2015. industry entitled ‘‘M3(R2) Nonclinical
state/province and country (if ADDRESSES: Submit written requests for Safety Studies for the Conduct of
provided), the name of your single copies of the draft guidance to the Human Clinical Trials and Marketing
representative (if any), and the category Division of Drug Information, Center for Authorization for Pharmaceuticals,’’
identifying you (e.g., individual, Drug Evaluation and Research, Food ‘‘M3(R2) Nonclinical Safety Studies for
asabaliauskas on DSK5VPTVN1PROD with NOTICES
consumer, academic, industry). For and Drug Administration, 10001 New the Conduct of Human Clinical Trials
written submissions submitted to the Hampshire Ave., Hillandale Building, and Marketing Authorization for
Division of Dockets Management, FDA 4th Floor, Silver Spring, MD 20993– Pharmaceuticals—Questions and
will post the body of your comments on 0002. Send one self-addressed adhesive Answers,’’ and ‘‘S6 Preclinical Safety
http://www.regulations.gov, but you can label to assist that office in processing Evaluation of Biotechnology-Derived
put your name and/or contact your requests. See the SUPPLEMENTARY Pharmaceuticals.’’
information on a separate cover sheet INFORMATION section for electronic This draft guidance is being issued
and not in the body of your comments. access to the draft guidance document. consistent with FDA’s good guidance
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![27320 Federal Register / Vol. 80, No. 92 / Wednesday, May 13, 2015 / Notices
practices regulation (21 CFR 10.115). Dated: May 7, 2015. contains the same active ingredient in
The draft guidance, when finalized, will Leslie Kux, the same strength and dosage form as
represent the current thinking of FDA Associate Commissioner for Policy. the ‘‘listed drug,’’ which is a version of
on nonclinical assessment of [FR Doc. 2015–11539 Filed 5–12–15; 8:45 am] the drug that was previously approved.
investigational ERT products. It does BILLING CODE 4164–01–P ANDA applicants do not have to repeat
not establish any rights for any person the extensive clinical testing otherwise
and is not binding on FDA or the public. necessary to gain approval of a new
You can use an alternative approach if DEPARTMENT OF HEALTH AND drug application (NDA).
it satisfies the requirements of the HUMAN SERVICES The 1984 amendments include what
applicable statutes and regulations. is now section 505(j)(7) of the Federal
Food and Drug Administration Food, Drug, and Cosmetic Act (21 U.S.C.
II. The Paperwork Reduction Act of [Docket No. FDA–2015–N–1339] 355(j)(7)), which requires FDA to
1995 publish a list of all approved drugs.
Determination That SODIUM SULAMYD FDA publishes this list as part of the
This guidance refers to previously (sulfacetamide sodium) Ophthalmic ‘‘Approved Drug Products with
approved collections of information that Solution and Ophthalmic Ointment Therapeutic Equivalence Evaluations,’’
are subject to review by the Office of Were Not Withdrawn From Sale for which is generally known as the
Management and Budget (OMB) under Reasons of Safety or Effectiveness ‘‘Orange Book.’’ Under FDA regulations,
the Paperwork Reduction Act of 1995 a drug is removed from the list if the
(44 U.S.C. 3501–3520). The collections AGENCY: Food and Drug Administration,
HHS. Agency withdraws or suspends
of information in 21 CFR part 312 have approval of the drug’s NDA or ANDA
been approved under OMB control ACTION: Notice.
for reasons of safety or effectiveness or
number 0910–0014, and the information SUMMARY: The Food and Drug if FDA determines that the listed drug
collection in the regulations on good Administration (FDA or Agency) has was withdrawn from sale for reasons of
laboratory practice for nonclinical determined that the drug products listed safety or effectiveness (21 CFR 314.162).
laboratory studies (21 CFR part 58) is in this document were not withdrawn Under § 314.161(a) (21 CFR
approved under OMB control number from sale for reasons of safety or 314.161(a)), the Agency must determine
0910–0119. effectiveness. This determination means whether a listed drug was withdrawn
that FDA will not begin procedures to from sale for reasons of safety or
III. Comments withdraw approval of abbreviated new effectiveness: (1) Before an ANDA that
Interested persons may submit either drug applications (ANDAs) that refer to refers to that listed drug may be
electronic comments regarding this these drug products, and it will allow approved, (2) whenever a listed drug is
FDA to continue to approve ANDAs that voluntarily withdrawn from sale and
document to http://www.regulations.gov
refer to the products as long as they ANDAs that refer to the listed drug have
or written comments to the Division of
meet relevant legal and regulatory been approved, and (3) when a person
Dockets Management (see ADDRESSES). It requirements.
is only necessary to send one set of petitions for such a determination under
comments. Identify comments with the FOR FURTHER INFORMATION CONTACT: 21 CFR 10.25(a) and 10.30. Section
docket number found in brackets in the Amy Hopkins, Center for Drug 314.161(d) provides that if FDA
Evaluation and Research, Food and determines that a listed drug was
heading of this document. Received
Drug Administration, 10903 New withdrawn from sale for safety or
comments may be seen in the Division
Hampshire Ave., Bldg. 51, Rm. 6223, effectiveness reasons, the Agency will
of Dockets Management between 9 a.m. initiate proceedings that could result in
Silver Spring, MD 20993–0002, 301–
and 4 p.m., Monday through Friday, and the withdrawal of approval of the
796–5418, Amy.Hopkins@fda.hhs.gov.
will be posted to the docket at http:// ANDAs that refer to the listed drug.
SUPPLEMENTARY INFORMATION: In 1984,
www.regulations.gov.
Congress enacted the Drug Price FDA has become aware that the drug
IV. Electronic Access Competition and Patent Term products listed in the table in this
Restoration Act of 1984 (Pub. L. 98–417) document are no longer being marketed.
Persons with access to the Internet (the 1984 amendments), which (As requested by the applicant, FDA
may obtain the document at either authorized the approval of duplicate withdrew approval of NDA 005963 for
http://www.fda.gov/Drugs/Guidance versions of drug products approved SODIUM SULAMYD (sulfacetamide
ComplianceRegulatoryInformation/ under an ANDA procedure. ANDA sodium) Ophthalmic Solution and
Guidances/default.htm or http:// applicants must, with certain Ophthalmic Ointment in the Federal
www.regulations.gov. exceptions, show that the drug for Register of August 16, 2001 (66 FR
which they are seeking approval 43017)).
Application No. Drug Applicant
NDA 005963 ........................ SODIUM SULAMYD (sulfacetamide sodium) Oph- Schering Plough Corp., 2000 Galloping Hill Rd., Ken-
thalmic Solution 10%, Ophthalmic Solution 30%, and ilworth, NJ 07033.
Ophthalmic Ointment 10%.
asabaliauskas on DSK5VPTVN1PROD with NOTICES
FDA has reviewed its records and, effectiveness. Accordingly, the Agency ‘‘Discontinued Drug Product List’’
under § 314.161, has determined that will continue to list the drug products identifies, among other items, drug
the drug products listed in this listed in this document in the products that have been discontinued
document were not withdrawn from ‘‘Discontinued Drug Product List’’ from marketing for reasons other than
sale for reasons of safety or section of the Orange Book. The safety or effectiveness.
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Document Created: 2015-12-16 07:46:59
Document Modified: 2015-12-16 07:46:59
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| 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. | |
| 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. | |
| FR Citation | 80 FR 27319 |
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