Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 32303 

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.gpo.gov/ 

fdsys/pkg/FR-2015-09-18/pdf/2015- 

23389.pdf. 

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copies of the draft guidance to the Office 

of Communication, Outreach and 

Development, Center for Biologics 

Evaluation and Research (CBER), 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 the office in processing your 

requests. The draft guidance may also be 

obtained by mail by calling CBER at 1– 

800–835–4709 or 240–402–8010. See 

the SUPPLEMENTARY INFORMATION section 

for electronic access to the draft 

guidance document. 

FOR FURTHER INFORMATION CONTACT: 

Angela Moy, 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 document entitled ‘‘Human Gene 

Therapy for Retinal Disorders; Draft 

Guidance for Industry.’’ The draft 

guidance provides recommendations to 

stakeholders developing GT products 

for retinal disorders affecting adult and 

pediatric patients. These disorders vary 

in etiology, prevalence, diagnosis, and 

management, and include genetic as 

well as age-related diseases. These 

disorders manifest with central or 

peripheral visual impairment and often 

with progressive visual loss. The draft 

guidance focuses on issues specific to 

GT products for retinal disorders and 

provides recommendations related to 

product development, preclinical 

testing, and clinical trial design for such 

GT products. 

Elsewhere in this issue of the Federal 

Register, FDA is announcing the 

availability of two other human gene 

therapy draft guidance documents 

entitled ‘‘Human Gene Therapy for 

Hemophilia; Draft Guidance for 

Industry’’ and ‘‘Human Gene Therapy 

for Rare Diseases; Draft Guidance for 

Industry.’’ 

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 ‘‘Human Gene Therapy for Retinal 

Disorders.’’ 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 draft guidance refers to 

previously approved collections of 

information 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 50 have been approved under OMB 

control number 0910–0755; the 

collections of information in 21 CFR 

part 58 have been approved under OMB 

control number 0910–0119; the 

collections of information in 21 CFR 

part 211 have been approved under 

OMB control number 0910–0139; 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 601 have been approved under 

OMB control number 0910–0338; the 

collections of information in the 

guidance entitled ‘‘Expedited Programs 

for Serious Conditions—Drugs and 

Biologics’’ have been approved under 

OMB control number 0910–0765; and 

the collections of information in the 

guidance entitled ‘‘Formal Meetings 

Between the FDA and Sponsors or 

Applicants’’ have been approved under 

OMB control number 0910–0429. 

III. Electronic Access 

Persons with access to the internet 

may obtain the draft guidance at either 

https://www.fda.gov/BiologicsBlood

Vaccines/GuidanceCompliance

RegulatoryInformation/Guidances/ 

default.htm or https://

www.regulations.gov. 

Dated: July 5, 2018. 

Leslie Kux, 

Associate Commissioner for Policy. 

[FR Doc. 2018–14870 Filed 7–11–18; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2018–D–2258] 

Human Gene Therapy for Rare 

Diseases; 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 

document entitled ‘‘Human Gene 

Therapy for Rare Diseases; Draft 

Guidance for Industry.’’ The draft 

guidance document provides 

recommendations to stakeholders 

developing a human gene therapy (GT) 

product intended to treat a rare disease 

in adult and/or pediatric patients 

regarding the manufacturing, 

preclinical, and clinical trial design 

issues for all phases of the clinical 

development program. Such 

information is intended to assist 

sponsors in designing clinical 

development programs for such 

products, where there may be limited 

study population size and potential 

feasibility and safety issues as well as 

issues relating to the interpretability of 

bioactivity/efficacy outcomes that may 

be unique to rare diseases or to the 

nature of the GT product itself. 

DATES: Submit either electronic or 

written comments on the draft guidance 

by October 10, 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 


amozie on DSK3GDR082PROD with NOTICES1
32304 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 

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–2258 for ‘‘Human Gene 

Therapy for Rare Diseases; Draft 

Guidance for Industry.’’ 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.gpo.gov/ 

fdsys/pkg/FR-2015-09-18/pdf/2015- 

23389.pdf. 

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 Office 

of Communication, Outreach and 

Development, Center for Biologics 

Evaluation and Research (CBER), 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 the office in processing your 

requests. The draft guidance may also be 

obtained by mail by calling CBER at 1– 

800–835–4709 or 240–402–8010. See 

the SUPPLEMENTARY INFORMATION section 

for electronic access to the draft 

guidance document. 

FOR FURTHER INFORMATION CONTACT: 

Jonathan McKnight, 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 

The Orphan Drug Act of 1983 (Pub. L. 

97–414) defines a rare disease as a 

disease or condition that affects fewer 

than 200,000 persons in the United 

States. Since most rare diseases have no 

approved therapies, there is a significant 

unmet need for effective treatments. 

However, developing safe and effective 

products to treat rare diseases can be 

challenging. For example, it may be 

more difficult to find and recruit such 

patients into clinical trials, and many 

rare diseases exhibit a number of 

variations or subtypes. Consequently, 

patients may have highly diverse 

clinical manifestations and rates of 

disease progression with unpredictable 

clinical courses. Despite these 

challenges, GT-related research and 

development continue to grow at a rapid 

rate, with several products advancing in 

clinical development. 

FDA is announcing the availability of 

a document entitled ‘‘Human Gene 

Therapy for Rare Diseases; Draft 

Guidance for Industry.’’ The draft 

guidance provides recommendations to 

stakeholders developing a GT product 

intended to treat a rare disease in adult 

and/or pediatric patients regarding the 

manufacturing, preclinical, and clinical 

trial design issues for all phases of the 

clinical development program. Such 

information is intended to assist 

sponsors in designing clinical 

development programs for such 

products, where there may be limited 

study population size and potential 

feasibility and safety issues as well as 

issues relating to the interpretability of 

bioactivity/efficacy outcomes that may 

be unique to rare diseases or to the 

nature of the GT product itself. 

Elsewhere in this issue of the Federal 

Register, FDA is announcing the 

availability of two other human gene 

therapy draft guidance documents 

entitled ‘‘Human Gene Therapy for 

Hemophilia; Draft Guidance for 

Industry’’ and ‘‘Human Gene Therapy 

for Retinal Disorders; Draft Guidance for 

Industry.’’ 

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 ‘‘Human Gene Therapy for Rare 

Diseases.’’ 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 draft guidance refers to 

previously approved collections of 

information 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 50 have been approved under OMB 

control number 0910–0755; the 


amozie on DSK3GDR082PROD with NOTICES1
Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 32305 

collections of information in 21 CFR 

part 58 have been approved under OMB 

control number 0910–0119; 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 601 have been approved under 

OMB control number 0910–0338; the 

collections of information in the 

guidance entitled ‘‘Expedited Programs 

for Serious Conditions—Drugs and 

Biologics’’ have been approved under 

OMB control number 0910–0765; and 

the collections of information in the 

guidance entitled ‘‘Formal Meetings 

Between the FDA and Sponsors or 

Applicants’’ have been approved under 

OMB control number 0910–0429. 

III. Electronic Access 

Persons with access to the internet 

may obtain the draft guidance at either 

https://www.fda.gov/BiologicsBlood

Vaccines/GuidanceCompliance

RegulatoryInformation/Guidances/ 

default.htm or https://

www.regulations.gov. 

Dated: July 5, 2018. 

Leslie Kux, 

Associate Commissioner for Policy. 

[FR Doc. 2018–14871 Filed 7–11–18; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2018–N–2180] 

Concordia Pharmaceuticals, Inc., et al.; 

Withdrawal of Approval of 29 New 

Drug Applications 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice. 

SUMMARY: The Food and Drug 

Administration (FDA or Agency) is 

withdrawing approval of 29 new drug 

applications (NDAs) from multiple 

applicants. The holders of the 

applications notified the Agency in 

writing that the drug products were no 

longer marketed and requested that the 

approval of the applications be 

withdrawn. 

DATES: Approval is withdrawn as of 

August 13, 2018. 

FOR FURTHER INFORMATION CONTACT: 

Florine P. Purdie, Center for Drug 

Evaluation and Research, Food and 

Drug Administration, 10903 New 

Hampshire Ave., Bldg. 51, Rm. 6248, 

Silver Spring, MD 20993–0002, 301– 

796–3601. 

SUPPLEMENTARY INFORMATION: The 

holders of the applications listed in the 

table have informed FDA that these drug 

products are no longer marketed and 

have requested that FDA withdraw 

approval of the applications under the 

process in § 314.150(c) (21 CFR 

314.150(c)). The applicants have also, 

by their requests, waived their 

opportunity for a hearing. Withdrawal 

of approval of an application or 

abbreviated application under 

§ 314.150(c) is without prejudice to 

refiling. 

Application No. Drug Applicant 

NDA 011287 ......... Kayexalate (sodium polystyrene sulfonate) Powder for Suspension, 

453.6 gram (g)/bottle.

Concordia Pharmaceuticals, Inc., c/o Mapi USA, Inc., 2343 Alexandria 

Dr., Lexington, KY 40504. 

NDA 012249 ......... Librium (chlordiazepoxide hydrochloride (HCl)) Capsules, 5 milligram 

(mg), 10 mg, and 25 mg.

Valeant Pharmaceuticals North America, LLC, 400 Somerset Corporate Blvd., Bridgewater, NJ 08807. 

NDA 016211 ......... Miochol (acetylcholine chloride) for Ophthalmic Solution, 20 mg/vial .... Novartis Pharmaceuticals Corp., One Health Pl., East Hanover, NJ 

07936. 

NDA 018674 ......... Metro I.V. (metronidazole) Injection, 500 mg/100 milliliter (mL) ............. B. Braun Medical, Inc., 901 Marcon Blvd., Allentown, PA 18109. 

NDA 018852 ......... Sulfamethoxazole and Trimethoprim Tablets USP, 400 mg; 80 mg ...... Watson Laboratories, Inc., Subsidiary of Teva Pharmaceuticals USA, 

Inc., 425 Privet Rd., Horsham, PA 19044. 

NDA 018854 ......... Sulfamethoxazole and Trimethoprim Tablets USP, 800 mg; 160 mg .... Do. 

NDA 018988 ......... Vasocidin (prednisolone sodium phosphate and sulfacetamide sodium) 

Ophthalmic Solution, equivalent to (EQ) 0.23% phosphate/10%.

Novartis Pharmaceuticals Corp. 

NDA 019844 ......... Isolyte H in Dextrose 5% in Plastic Container Injection ......................... B. Braun Medical, Inc. 

NDA 019870 ......... Isolyte M in Dextrose 5% in Plastic Container Injection ......................... Do. 

NDA 019964 ......... Terazol 3 (terconazole) Vaginal Cream, 0.8% ........................................ Janssen Pharmaceuticals, Inc., 1125 Trenton-Harbourton Rd., 

Titusville, NJ 08560. 

NDA 020000 ......... Dextrose 5% in Ringer’s in Plastic Container Injection .......................... B. Braun Medical, Inc. 

NDA 020393 ......... Atrovent (ipratropium bromide) Nasal Spray, 0.021 mg/spray ............... Boehringer Ingelheim Pharmaceuticals, Inc., 900 Ridgebury Rd., P.O. 

Box 368, Ridgefield, CT 06877–0368. 

NDA 020394 ......... Atrovent (ipratropium bromide) Nasal Spray, 0.042 mg/spray ............... Do. 

NDA 021180 ......... Ortho Evra (ethinyl estradiol; norelgestromin) Transdermal Patch, 

0.035 mg/24 h; 0.15 mg/24 h.

Janssen Pharmaceuticals, Inc., 1000 U.S. Route 202, P.O. Box 300, 

Raritan, NJ 08869–0602. 

NDA 021633 ......... Femtrace (estradiol acetate) Tablets, 0.45 mg, 0.9 mg, and 1.8 mg ..... Allergan Pharmaceuticals International, Ltd., c/o Allergan Sales, LLC, 

2525 Dupont Dr., Irvine, CA 92612. 

NDA 022033 ......... Luvox CR (fluvoxamine maleate) Extended-Release Capsules, 100 mg 

and 150 mg.

Jazz Pharmaceuticals, Inc., 3180 Porter Dr., Palo Alto, CA 94304. 

NDA 022106 ......... Doribax (doripenem) for Injection, 250 mg/vial and 500 mg/vial ............ Shionogi, Inc., 300 Campus Dr., Florham Park, NJ 07932. 

NDA 022386 ......... PrandiMet (metformin HCl; repaglinide) Tablets, 500mg; 1 mg and 500 

mg; 2 mg.

Novo Nordisk, Inc., P.O. Box 846, Plainsboro, NJ 08536. 

NDA 050201 ......... Ophthocort (chloramphenicol, hydrocortisone acetate, polymyxin B sulfate) Ophthalmic Ointment USP, 10 mg/g; 5 mg/g; 10,000 units/g.Parkedale Pharmaceuticals, Subsidiary of Pfizer Inc., 235 East 42nd 

St., New York, NY 10017. 

NDA 050344 ......... Statrol (neomycin sulfate; polymyxin B sulfate) Ophthalmic Ointment, 

EQ 3.5 mg base/g; 10,000 units/g.

Alcon Laboratories, Inc., 6201 South Freeway, TC–45, Fort Worth, TX 

76134. 

NDA 050442 ......... Vibramycin (doxycycline hyclate) Injection, EQ to 200 mg base/vial 

and EQ 100 mg base/vial.

Pfizer, Inc., 235 East 42nd St., New York, NY 10017. 

NDA 050497 ......... Ticar (ticarcillin disodium) Injection, EQ 1 g base/vial, EQ 3 g base/ 

vial, EQ 6 g base/vial, EQ 20 g base/vial, and EQ 30 g base/vial.

GlaxoSmithKline, 1250 Collegeville Rd., Collegeville, PA 19426. 

NDA 050512 ......... Duricef (cefadroxil monohydrate) USP Capsules, EQ 500 mg base and 

EQ 250 mg base.

Warner Chilcott Co., LLC, 100 Enterprise Dr., Rockaway, NJ 07866. 

NDA 050527 ......... Duricef (cefadroxil monohydrate) USP For Oral Suspension, EQ 125 

mg base/5 mL, EQ 250 mg base/5 mL, and EQ 500 mg base/5 mL.

Do. 

NDA 050593 ......... Eryc Sprinkles (erythromycin) Capsules, 125 mg ................................... Hospira Inc., 275 North Field Dr., Lake Forest, IL 60045. 

NDA 050646 ......... Ceptaz (ceftazidime) Injection, 500 mg/vial, 1 g/vial, 2 g/vial, and 10 g/ 

vial.

GlaxoSmithKline. 

NDA 050668 ......... Lorabid (loracarbef) Capsules USP, 200 mg and 400 mg ..................... King Pharmaceuticals, Inc., 501 Fifth St., Bristol, TN 37620. 

NDA 050792 ......... Cefotaxime and Dextrose 2.4% in Plastic Container, EQ 2 g base, and 

Cefotaxime and Dextrose 3.9% in Plastic Container, EQ 1 g base.

B. Braun Medical, Inc. 


amozie on DSK3GDR082PROD with NOTICES1