83 FR 35653 - Slowly Progressive, Low-Prevalence Rare Diseases With Substrate Deposition That Results From Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 145 (July 27, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 145 (July 27, 2018)
| Page Range | 35653-35654 | |
| FR Document | 2018-16036 |
[Federal Register Volume 83, Number 145 (Friday, July 27, 2018)] [Notices] [Pages 35653-35654] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-16036] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2018-D-2456] Slowly Progressive, Low-Prevalence Rare Diseases With Substrate Deposition That Results From Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies.'' This document is intended to provide guidance to sponsors on the evidence necessary to demonstrate the effectiveness of new drugs, including biological drugs, or new drug uses intended for slowly progressive, low-prevalence rare diseases that are associated with substrate deposition and are caused by single enzyme defects. This guidance applies only to those low-prevalence rare diseases with a well-characterized pathophysiology and in which changes in substrate deposition can be readily measured in relevant tissue(s). DATES: Submit either electronic or written comments on the draft guidance by September 25, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. ADDRESSES: You may submit comments on any guidance at any time as follows: Electronic Submissions Submit electronic comments in the following way:Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else's Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov. If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see ``Written/Paper Submissions'' and ``Instructions''). Written/Paper Submissions Submit written/paper submissions as follows: Mail/Hand delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ``Instructions.'' Instructions: All submissions received must include the Docket No. FDA-2018-D-2456 for ``Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies; Draft Guidance for Industry; Availability.'' Received comments will be placed in the docket and, except for those submitted as ``Confidential Submissions,'' publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday. Confidential Submissions--To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as ``confidential.'' Any information marked as ``confidential'' will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA's posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.thefederalregister.org/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf. [[Page 35654]] Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ``Search'' box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)). 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, or Office of Communication, Outreach, and Development, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, 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. FOR FURTHER INFORMATION CONTACT: Dragos Roman, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 5152, Silver Spring, MD 20993-0002, 301- 796-1285; or Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ``Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies.'' This document is intended to provide guidance to sponsors on the evidence necessary to demonstrate the effectiveness of new drugs or new drug uses intended for slowly progressive, low-prevalence rare diseases that are associated with substrate deposition and are caused by single enzyme defects. This guidance applies only to those low-prevalence rare diseases with a well-characterized pathophysiology and in which changes in substrate deposition can be readily measured in relevant tissue(s). This draft guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on providing evidence of effectiveness for replacement or corrective therapies intended for slowly progressive, low-prevalence rare diseases with substrate deposition that results from single enzyme defects. 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. This guidance is not subject to Executive Order 12866. II. 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. The collections of information in 21 CFR part 50 have been approved under OMB control number 0910-0755. The collections of information for expedited programs in the guidance for industry entitled ``Expedited Programs for Serious Conditions--Drugs and Biologics'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm358301.pdf) have been approved under OMB control number 0910-0765. III. Electronic Access Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or https://www.regulations.gov. Dated: July 20, 2018. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2018-16036 Filed 7-26-18; 8:45 am] BILLING CODE 4164-01-P
![Federal Register / Vol. 83, No. 145 / Friday, July 27, 2018 / Notices 35653
Cover Sheet and generating the user fee ACTION: Notice of availability. Written/Paper Submissions
ID number. Submit written/paper submissions as
The user fee ID number must be SUMMARY: The Food and Drug follows:
included on the check, bank draft, or Administration (FDA or Agency) is • Mail/Hand delivery/Courier (for
postal money order and must be made announcing the availability of a draft written/paper submissions): Dockets
payable to the order of the Food and guidance for industry entitled ‘‘Slowly Management Staff (HFA–305), Food and
Drug Administration. Payments can be Progressive, Low-Prevalence Rare Drug Administration, 5630 Fishers
mailed to: Food and Drug Diseases with Substrate Deposition That Lane, Rm. 1061, Rockville, MD 20852.
Administration, P.O. Box 979108, St. Results from Single Enzyme Defects: • For written/paper comments
Louis, MO 63197–9000. If checks are to Providing Evidence of Effectiveness for submitted to the Dockets Management
be sent by a courier that requests a street Replacement or Corrective Therapies.’’ Staff, FDA will post your comment, as
address, the courier can deliver checks This document is intended to provide well as any attachments, except for
to: U.S. Bank, Attention: Government guidance to sponsors on the evidence information submitted, marked and
Lockbox 979108, 1005 Convention necessary to demonstrate the identified, as confidential, if submitted
Plaza, St. Louis, MO 63101. (Note: This effectiveness of new drugs, including as detailed in ‘‘Instructions.’’
U.S. Bank address is for courier delivery biological drugs, or new drug uses Instructions: All submissions received
only. For questions concerning courier intended for slowly progressive, low- must include the Docket No. FDA–
delivery, U.S. Bank can be contacted at prevalence rare diseases that are 2018–D–2456 for ‘‘Slowly Progressive,
314–418–4013. This telephone number associated with substrate deposition and Low-Prevalence Rare Diseases with
is only for questions about courier are caused by single enzyme defects. Substrate Deposition That Results from
delivery.) The FDA post office box This guidance applies only to those low- Single Enzyme Defects: Providing
number (P.O. Box 979108) must be prevalence rare diseases with a well- Evidence of Effectiveness for
written on the check, bank draft, or characterized pathophysiology and in Replacement or Corrective Therapies;
postal money order. which changes in substrate deposition Draft Guidance for Industry;
For payments made by wire transfer, can be readily measured in relevant Availability.’’ Received comments will
the unique user fee ID number must be tissue(s). be placed in the docket and, except for
referenced. Without the unique user fee those submitted as ‘‘Confidential
ID number, the payment may not be DATES: Submit either electronic or Submissions,’’ publicly viewable at
applied. If the payment amount is not written comments on the draft guidance https://www.regulations.gov or at the
applied, the invoice amount will be by September 25, 2018 to ensure that Dockets Management Staff between 9
referred to collections. The originating the Agency considers your comment on a.m. and 4 p.m., Monday through
financial institution may charge a wire this draft guidance before it begins work Friday.
transfer fee. Applicable wire transfer on the final version of the guidance. • Confidential Submissions—To
fees must be included with payment to ADDRESSES: You may submit comments submit a comment with confidential
ensure fees are fully paid. Questions on any guidance at any time as follows: information that you do not wish to be
about wire transfer fees should be made publicly available, submit your
addressed to the financial institution. Electronic Submissions comments only as a written/paper
The following account information submission. You should submit two
should be used to send payments by Submit electronic comments in the
following way: copies total. One copy will include the
wire transfer: U.S. Department of information you claim to be confidential
Treasury, TREAS NYC, 33 Liberty St., • Federal eRulemaking Portal: with a heading or cover note that states
New York, NY 10045, account number: https://www.regulations.gov. Follow the ‘‘THIS DOCUMENT CONTAINS
75060099, routing number: 021030004, instructions for submitting comments. CONFIDENTIAL INFORMATION.’’ The
SWIFT: FRNYUS33. FDA’s tax Comments submitted electronically, Agency will review this copy, including
identification number is 53–0196965. including attachments, to https:// the claimed confidential information, in
Dated: July 24, 2018.
www.regulations.gov will be posted to its consideration of comments. The
the docket unchanged. Because your second copy, which will have the
Leslie Kux,
comment will be made public, you are claimed confidential information
Associate Commissioner for Policy. solely responsible for ensuring that your
[FR Doc. 2018–16067 Filed 7–26–18; 8:45 am]
redacted/blacked out, will be available
comment does not include any for public viewing and posted on
BILLING CODE 4164–01–P confidential information that you or a https://www.regulations.gov. Submit
third party may not wish to be posted, both copies to the Dockets Management
such as medical information, your or Staff. If you do not wish your name and
DEPARTMENT OF HEALTH AND anyone else’s Social Security number, or
HUMAN SERVICES contact information to be made publicly
confidential business information, such available, you can provide this
as a manufacturing process. Please note information on the cover sheet and not
Food and Drug Administration
that if you include your name, contact in the body of your comments and you
[Docket No. FDA–2018–D–2456] information, or other information that must identify this information as
identifies you in the body of your ‘‘confidential.’’ Any information marked
Slowly Progressive, Low-Prevalence comments, that information will be
Rare Diseases With Substrate as ‘‘confidential’’ will not be disclosed
posted on https://www.regulations.gov. except in accordance with 21 CFR 10.20
amozie on DSK3GDR082PROD with NOTICES1
Deposition That Results From Single
Enzyme Defects: Providing Evidence • If you want to submit a comment and other applicable disclosure law. For
of Effectiveness for Replacement or with confidential information that you more information about FDA’s posting
Corrective Therapies; Draft Guidance do not wish to be made available to the of comments to public dockets, see 80
for Industry; Availability public, submit the comment as a FR 56469, September 18, 2015, or access
written/paper submission and in the the information at: https://www.gpo.gov/
AGENCY: Food and Drug Administration, manner detailed (see ‘‘Written/Paper fdsys/pkg/FR-2015-09-18/pdf/2015-
HHS. Submissions’’ and ‘‘Instructions’’). 23389.pdf.
VerDate Sep<11>2014 17:38 Jul 26, 2018 Jkt 244001 PO 00000 Frm 00050 Fmt 4703 Sfmt 4703 E:\FR\FM\27JYN1.SGM 27JYN1](https://thefederalregister.org/doc/83_FR_35798/page/1.svg)
![35654 Federal Register / Vol. 83, No. 145 / Friday, July 27, 2018 / Notices
Docket: For access to the docket to drugs or new drug uses intended for Information/default.htm, or https://
read background documents or the slowly progressive, low-prevalence rare www.regulations.gov.
electronic and written/paper comments diseases that are associated with Dated: July 20, 2018.
received, go to https:// substrate deposition and are caused by Leslie Kux,
www.regulations.gov and insert the single enzyme defects. This guidance
Associate Commissioner for Policy.
docket number, found in brackets in the applies only to those low-prevalence
rare diseases with a well-characterized [FR Doc. 2018–16036 Filed 7–26–18; 8:45 am]
heading of this document, into the
‘‘Search’’ box and follow the prompts pathophysiology and in which changes BILLING CODE 4164–01–P
and/or go to the Dockets Management in substrate deposition can be readily
Staff, 5630 Fishers Lane, Rm. 1061, measured in relevant tissue(s).
This draft guidance is being issued DEPARTMENT OF HEALTH AND
Rockville, MD 20852.
You may submit comments on any consistent with FDA’s good guidance HUMAN SERVICES
guidance at any time (see 21 CFR practices regulation (21 CFR 10.115).
Food and Drug Administration
10.115(g)(5)). The draft guidance, when finalized, will
Submit written requests for single represent the current thinking of FDA
copies of the draft guidance to the on providing evidence of effectiveness [Docket No. FDA–2018–N–2493]
Division of Drug Information, Center for for replacement or corrective therapies
intended for slowly progressive, low- ICU Medical, Inc., et al.; Withdrawal of
Drug Evaluation and Research, Food
prevalence rare diseases with substrate Approval of 31 Abbreviated New Drug
and Drug Administration, 10001 New
deposition that results from single Applications
Hampshire Ave., Hillandale Building,
4th Floor, Silver Spring, MD 20993– enzyme defects. It does not establish AGENCY: Food and Drug Administration,
0002, or Office of Communication, any rights for any person and is not HHS.
Outreach, and Development, Center for binding on FDA or the public. You can
use an alternative approach if it satisfies ACTION: Notice.
Biologics Evaluation and Research,
Food and Drug Administration, 10903 the requirements of the applicable SUMMARY: The Food and Drug
New Hampshire Ave., Bldg. 71, Rm. statutes and regulations. This guidance Administration (FDA or Agency) is
3128, Silver Spring, MD 20993–0002. is not subject to Executive Order 12866. withdrawing approval of 31 abbreviated
Send one self-addressed adhesive label II. Paperwork Reduction Act of 1995 new drug applications (ANDAs) from
to assist that office in processing your multiple applicants. The holders of the
requests. See the SUPPLEMENTARY This guidance refers to previously
approved collections of information that applications notified the Agency in
INFORMATION section for electronic writing that the drug products were no
are subject to review by the Office of
access to the draft guidance document. longer marketed and requested that the
Management and Budget (OMB) under
FOR FURTHER INFORMATION CONTACT: approval of the applications be
the Paperwork Reduction Act of 1995
Dragos Roman, Center for Drug (44 U.S.C. 3501–3520). The collections withdrawn.
Evaluation and Research, Food and of information in 21 CFR part 312 have DATES: Approval is withdrawn as of
Drug Administration, 10903 New been approved under OMB control August 27, 2018.
Hampshire Ave., Bldg. 22, Rm. 5152, number 0910–0014. The collections of
Silver Spring, MD 20993–0002, 301– FOR FURTHER INFORMATION CONTACT:
information in 21 CFR part 50 have been Trang Tran, Center for Drug Evaluation
796–1285; or Stephen Ripley, Center for approved under OMB control number
Biologics Evaluation and Research, and Research, Food and Drug
0910–0755. The collections of
Food and Drug Administration, 10903 Administration, 10903 New Hampshire
information for expedited programs in
New Hampshire Ave., Bldg. 71, Rm. Ave., Bldg. 75, Rm. 1671, Silver Spring,
the guidance for industry entitled
7301, Silver Spring, MD 20993–0002, MD 20993–0002, 240–402–7945,
‘‘Expedited Programs for Serious
240–402–7911. Trang.Tran@fda.hhs.gov.
Conditions—Drugs and Biologics’’
SUPPLEMENTARY INFORMATION: (available at https://www.fda.gov/ucm/ SUPPLEMENTARY INFORMATION: The
groups/fdagov-public/@fdagov-drugs- holders of the applications listed in the
I. Background table have informed FDA that these drug
gen/documents/document/ucm35
FDA is announcing the availability of 8301.pdf) have been approved under products are no longer marketed and
a draft guidance for industry entitled OMB control number 0910–0765. have requested that FDA withdraw
‘‘Slowly Progressive, Low-Prevalence approval of the applications under the
Rare Diseases with Substrate Deposition III. Electronic Access process described in § 314.150(c) (21
That Results from Single Enzyme Persons with access to the internet CFR 314.150(c)). The applicants have
Defects: Providing Evidence of may obtain the draft guidance at either also, by their requests, waived their
Effectiveness for Replacement or https://www.fda.gov/Drugs/Guidance opportunity for a hearing. Withdrawal
Corrective Therapies.’’ This document is ComplianceRegulatoryInformation/ of approval of an application or
intended to provide guidance to Guidances/default.htm, http:// abbreviated application under
sponsors on the evidence necessary to www.fda.gov/BiologicsBloodVaccines/ § 314.150(c) is without prejudice to
demonstrate the effectiveness of new GuidanceComplianceRegulatory refiling.
Application No. Drug Applicant
amozie on DSK3GDR082PROD with NOTICES1
ANDA 020345 .... Aminosyn-HF (amino acids) Injection, 8% ............................... ICU Medical, Inc., 600 North Field Dr., Lake Forest, IL
60045.
ANDA 040723 .... Isosorbide Dinitrate Extended-Release Tablets USP, 40 milli- Impax Laboratories, Inc., 30831 Huntwood Ave., Hayward,
grams (mg). CA 94544.
ANDA 064062 .... Amphotericin B for Injection USP, 50 mg/vial ......................... Teva Parenteral Medicines, Inc., 19 Hughes, Irvine, CA
92618.
ANDA 064200 .... Cefotaxime for Injection USP, Equivalent to (EQ) 500 mg Fresenius Kabi USA, LLC, Three Corporate Dr., Lake Zurich,
base/vial, EQ 1 gram (g) base/vial, and EQ 2 g base/vial. IL 60047.
VerDate Sep<11>2014 17:38 Jul 26, 2018 Jkt 244001 PO 00000 Frm 00051 Fmt 4703 Sfmt 4703 E:\FR\FM\27JYN1.SGM 27JYN1](https://thefederalregister.org/doc/83_FR_35798/page/2.svg)
Document Created: 2018-07-27 04:05:25
Document Modified: 2018-07-27 04:05:25
| 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 of availability. | |
| Dates | Submit either electronic or written comments on the draft guidance by September 25, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. | |
| Contact | Dragos Roman, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 5152, Silver Spring, MD 20993-0002, 301- 796-1285; or Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911. | |
| FR Citation | 83 FR 35653 |
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