82_FR_59862 82 FR 59622 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product

82 FR 59622 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 82, Issue 240 (December 15, 2017)

Page Range59622-59623
FR Document2017-27049

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the Food and Drug Administration Safety and Innovation Act (FDASIA), authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that MEPSEVII (vestronidase alfa-vjbk), manufactured by Ultragenyx Pharmaceutical, Inc., meets the criteria for a priority review voucher.

Federal Register, Volume 82 Issue 240 (Friday, December 15, 2017) 
[Federal Register Volume 82, Number 240 (Friday, December 15, 2017)]
[Notices]
[Pages 59622-59623]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2017-27049]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0809]


Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
issuance of a priority review voucher to

[[Page 59623]]

the sponsor of a rare pediatric disease product application. The 
Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the 
Food and Drug Administration Safety and Innovation Act (FDASIA), 
authorizes FDA to award priority review vouchers to sponsors of 
approved rare pediatric disease product applications that meet certain 
criteria. FDA is required to publish notice of the award of the 
priority review voucher. FDA has determined that MEPSEVII (vestronidase 
alfa-vjbk), manufactured by Ultragenyx Pharmaceutical, Inc., meets the 
criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Althea Cuff, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301-
796-9856, email: [email protected].

SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority 
review voucher to the sponsor of an approved rare pediatric disease 
product application. Under section 529 of the FD&C Act (21 U.S.C. 
360ff), which was added by FDASIA, FDA will award priority review 
vouchers to sponsors of approved rare pediatric disease product 
applications that meet certain criteria. FDA has determined that 
MEPSEVII (vestronidase alfa-vjbk), manufactured by Ultragenyx 
Pharmaceutical, Inc., meets the criteria for a priority review voucher. 
MEPSEVII (vestronidase alfa-vjbk) is indicated for the treatment of 
Mucopolysaccharidosis type VII (MPS VII, Sly Syndrome).
    For further information about the Rare Pediatric Disease Priority 
Review Voucher Program and for a link to the full text of section 529 
of the FD&C Act, go to https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further 
information about MEPSEVII (vestronidase alfa-vjbk), go to the 
``Drugs@FDA'' website at https://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: December 12, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-27049 Filed 12-14-17; 8:45 am]
 BILLING CODE 4164-01-P

59622 Federal Register / Vol. 82, No. 240 / Friday, December 15, 2017 / Notices DEPARTMENT OF HEALTH AND proposed collection(s) summarized in coverage must satisfy in order to qualify HUMAN SERVICES this notice, you may make your request as excepted benefits. One of them is a using one of following: reporting requirement, for group health Centers for Medicare & Medicaid 1. Access CMS’ website address at plans and group health insurance Services http://www.cms.hhs.gov/ issuers, as well as group health plan [Document Identifier: CMS–10571] PaperworkReductionActof1995. sponsors. 2. Email your request, including your A self-insured group health plan, or a Agency Information Collection address, phone number, OMB number, health insurance issuer offering or Activities: Proposed Collection; and CMS document identifier, to proposing to offer Multi-State Plan Comment Request Paperwork@cms.hhs.gov. wraparound coverage, is required to 3. Call the Reports Clearance Office at report to OPM information reasonably AGENCY: Centers for Medicare & (410) 786–1326. required to determine whether the plan Medicaid Services, HHS. FOR FURTHER INFORMATION CONTACT: or issuer qualifies to offer such coverage ACTION: Notice. William Parham at (410) 786–4669. or complies with the applicable SUPPLEMENTARY INFORMATION: requirements. In addition, the plan SUMMARY: The Centers for Medicare & sponsor of any group health plan Medicaid Services (CMS) is announcing Contents offering any type of limited wraparound an opportunity for the public to This notice sets out a summary of the coverage is required to report to the comment on CMS’ intention to collect use and burden associated with the Department of Health and Human information from the public. Under the following information collections. More Services (HHS), in a form and manner Paperwork Reduction Act of 1995 (the detailed information can be found in specified in guidance by the Secretary of PRA), federal agencies are required to each collection’s supporting statement HHS. publish notice in the Federal Register and associated materials (see We seek comment on the content of concerning each proposed collection of ADDRESSES). the proposed collection form. We also information (including each proposed CMS–10571 Limited Wraparound seek comment on the impact that an extension or reinstatement of an existing Coverage Reporting extension of the limited wraparound collection of information) and to allow pilot program would have on the 60 days for public comment on the Under the PRA (44 U.S.C. 3501– number of employers/sponsors proposed action. Interested persons are 3520), federal agencies must obtain participating in the limited wraparound invited to send comments regarding our approval from the Office of Management pilot program. In addition, if HHS burden estimates or any other aspect of and Budget (OMB) for each collection of extends the limited wraparound pilot this collection of information, including information they conduct or sponsor. program, we seek comment on when the the necessity and utility of the proposed The term ‘‘collection of information’’ is limited wraparound pilot program information collection for the proper defined in 44 U.S.C. 3502(3) and 5 CFR should sunset, or whether the limited performance of the agency’s functions, 1320.3(c) and includes agency requests wraparound pilot program should be the accuracy of the estimated burden, or requirements that members of the made permanent. Form Number: CMS– ways to enhance the quality, utility, and public submit reports, keep records, or 10571 (OMB control number: 0938– clarity of the information to be provide information to a third party. NEW); Frequency: Once; Affected collected, and the use of automated Section 3506(c)(2)(A) of the PRA Public: Private Sector; Number of collection techniques or other forms of requires federal agencies to publish a Respondents: 8; Number of Responses: information technology to minimize the 60-day notice in the Federal Register 8; Total Annual Hours: 24. (For policy information collection burden. concerning each proposed collection of questions regarding this collection DATES: Comments must be received by information, including each proposed contact Usree Bandyopadhyay at 410– February 13, 2018. extension or reinstatement of an existing 786–6650). ADDRESSES: When commenting, please collection of information, before Dated: December 12, 2017. reference the document identifier or submitting the collection to OMB for OMB control number. To be assured approval. To comply with this William N. Parham, III, consideration, comments and requirement, CMS is publishing this Director, Paperwork Reduction Staff, Office recommendations must be submitted in notice. of Strategic Operations and Regulatory Affairs. any one of the following ways: Information Collection 1. Electronically. You may send your [FR Doc. 2017–27048 Filed 12–14–17; 8:45 am] comments electronically to http:// 1. Type of Information Collection BILLING CODE 4120–01–P www.regulations.gov. Follow the Request: New collection of information instructions for ‘‘Comment or request; Title of Information Collection: Submission’’ or ‘‘More Search Options’’ Limited Wraparound Coverage DEPARTMENT OF HEALTH AND to find the information collection Reporting; Use: The Department of HUMAN SERVICES document(s) that are accepting Treasury, the Department of Labor and Food and Drug Administration comments. the Department of Health and Human 2. By regular mail. You may mail Services published final regulations on [Docket No. FDA–2017–N–0809] written comments to the following March 18, 2015 (80 FR 13995), address: CMS, Office of Strategic amending the regulations regarding Issuance of Priority Review Voucher; excepted benefits under the Employee Rare Pediatric Disease Product sradovich on DSK3GMQ082PROD with NOTICES Operations and Regulatory Affairs, Division of Regulations Development, Retirement Income Security Act of 1974, AGENCY: Food and Drug Administration, Attention: Document Identifier/OMB the Internal Revenue Code, and the HHS. Control Number ll, Room C4–26–05, Public Health Service Act to specify ACTION: Notice. 7500 Security Boulevard, Baltimore, requirements for limited wraparound Maryland 21244–1850. coverage to qualify as an excepted SUMMARY: The Food and Drug To obtain copies of a supporting benefit. The final regulations include Administration (FDA) is announcing the statement and any related forms for the requirements that limited wraparound issuance of a priority review voucher to VerDate Sep<11>2014 23:42 Dec 14, 2017 Jkt 244001 PO 00000 Frm 00048 Fmt 4703 Sfmt 4703 E:\FR\FM\15DEN1.SGM 15DEN1

Federal Register / Vol. 82, No. 240 / Friday, December 15, 2017 / Notices 59623 the sponsor of a rare pediatric disease DEPARTMENT OF HEALTH AND comments, that information will be product application. The Federal Food, HUMAN SERVICES posted on https://www.regulations.gov. Drug, and Cosmetic Act (the FD&C Act), • If you want to submit a comment as amended by the Food and Drug Food and Drug Administration with confidential information that you Administration Safety and Innovation [Docket No. FDA–2017–D–6702] do not wish to be made available to the Act (FDASIA), authorizes FDA to award public, submit the comment as a priority review vouchers to sponsors of The Least Burdensome Provisions: written/paper submission and in the approved rare pediatric disease product Concept and Principles; Draft manner detailed (see ‘‘Written/Paper Guidance for Industry and Food and Submissions’’ and ‘‘Instructions’’). applications that meet certain criteria. FDA is required to publish notice of the Drug Administration Staff; Availability Written/Paper Submissions award of the priority review voucher. AGENCY: Food and Drug Administration, Submit written/paper submissions as FDA has determined that MEPSEVII HHS. follows: (vestronidase alfa-vjbk), manufactured ACTION: Notice of availability. • Mail/Hand delivery/Courier (for by Ultragenyx Pharmaceutical, Inc., written/paper submissions): Dockets meets the criteria for a priority review SUMMARY: The Food and Drug Management Staff (HFA–305), Food and voucher. Administration (FDA or Agency) is Drug Administration, 5630 Fishers announcing the availability of the draft Lane, Rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: guidance entitled ‘‘The Least • For written/paper comments Althea Cuff, Center for Drug Evaluation Burdensome Provisions: Concept and submitted to the Dockets Management and Research, Food and Drug Principles’’ FDA utilizes a least Staff, FDA will post your comment, as Administration, 10903 New Hampshire burdensome approach to medical device well as any attachments, except for Ave., Silver Spring, MD 20993–0002, regulation to eliminate unnecessary information submitted, marked and 301–796–4061, Fax: 301–796–9856, burdens that may delay the marketing of identified, as confidential, if submitted email: althea.cuff@fda.hhs.gov. beneficial new products, while as detailed in ‘‘Instructions.’’ maintaining the statutory requirements Instructions: All submissions received SUPPLEMENTARY INFORMATION: FDA is for clearance and approval. This must include the Docket No. FDA– announcing the issuance of a priority document describes the guiding 2017–D–6702 for ‘‘The Least review voucher to the sponsor of an principles and recommended approach Burdensome Provisions: Concept and approved rare pediatric disease product for FDA staff and industry to facilitate Principles; Draft Guidance for Industry application. Under section 529 of the consistent application of least and Food and Drug Administration FD&C Act (21 U.S.C. 360ff), which was burdensome principles to the activities Staff; Availability.’’ Received comments added by FDASIA, FDA will award pertaining to products meeting the will be placed in the docket and, except priority review vouchers to sponsors of statutory definition of a device regulated for those submitted as ‘‘Confidential approved rare pediatric disease product under the Federal Food, Drug, and Submissions,’’ publicly viewable at applications that meet certain criteria. Cosmetic Act (the FD&C Act). This draft https://www.regulations.gov or at the FDA has determined that MEPSEVII guidance is not final nor is it in effect Dockets Management Staff between 9 (vestronidase alfa-vjbk), manufactured at this time. a.m. and 4 p.m., Monday through by Ultragenyx Pharmaceutical, Inc., DATES: Submit either electronic or Friday. meets the criteria for a priority review written comments on the draft guidance • Confidential Submissions—To voucher. MEPSEVII (vestronidase alfa- by February 13, 2018 to ensure that the submit a comment with confidential vjbk) is indicated for the treatment of Agency considers your comment on this information that you do not wish to be Mucopolysaccharidosis type VII (MPS draft guidance before it begins work on made publicly available, submit your VII, Sly Syndrome). the final version of the guidance. comments only as a written/paper ADDRESSES: You may submit comments submission. You should submit two For further information about the Rare copies total. One copy will include the Pediatric Disease Priority Review on any guidance at any time as follows: information you claim to be confidential Voucher Program and for a link to the Electronic Submissions with a heading or cover note that states full text of section 529 of the FD&C Act, Submit electronic comments in the ‘‘THIS DOCUMENT CONTAINS go to https://www.fda.gov/ForIndustry/ following way: CONFIDENTIAL INFORMATION.’’ The DevelopingProductsforRareDiseases • Federal eRulemaking Portal: Agency will review this copy, including Conditions/RarePediatricDiseasePriority https://www.regulations.gov. Follow the the claimed confidential information, in VoucherProgram/default.htm. For instructions for submitting comments. its consideration of comments. The further information about MEPSEVII Comments submitted electronically, second copy, which will have the (vestronidase alfa-vjbk), go to the including attachments, to https:// claimed confidential information ‘‘Drugs@FDA’’ website at https://www. www.regulations.gov will be posted to redacted/blacked out, will be available accessdata.fda.gov/scripts/cder/daf/. the docket unchanged. Because your for public viewing and posted on comment will be made public, you are https://www.regulations.gov. Submit Dated: December 12, 2017. solely responsible for ensuring that your both copies to the Dockets Management Leslie Kux, comment does not include any Staff. If you do not wish your name and Associate Commissioner for Policy. confidential information that you or a contact information to be made publicly third party may not wish to be posted, available, you can provide this sradovich on DSK3GMQ082PROD with NOTICES [FR Doc. 2017–27049 Filed 12–14–17; 8:45 am] BILLING CODE 4164–01–P such as medical information, your or information on the cover sheet and not anyone else’s Social Security number, or in the body of your comments and you confidential business information, such must identify this information as as a manufacturing process. Please note ‘‘confidential.’’ Any information marked that if you include your name, contact as ‘‘confidential’’ will not be disclosed information, or other information that except in accordance with 21 CFR 10.20 identifies you in the body of your and other applicable disclosure law. For VerDate Sep<11>2014 23:42 Dec 14, 2017 Jkt 244001 PO 00000 Frm 00049 Fmt 4703 Sfmt 4703 E:\FR\FM\15DEN1.SGM 15DEN1


Document Created: 2017-12-15 03:37:12
Document Modified: 2017-12-15 03:37:12
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice.
ContactAlthea Cuff, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301- 796-9856, email: [email protected]
FR Citation82 FR 59622 
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