80 FR 18628 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 66 (April 7, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 66 (April 7, 2015)
| Page Range | 18628-18628 | |
| FR Document | 2015-08016 |
[Federal Register Volume 80, Number 66 (Tuesday, April 7, 2015)] [Notices] [Page 18628] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2015-08016] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2014-N-0229] Issuance of Priority Review Voucher; Rare Pediatric Disease Product AGENCY: Food and Drug Administration, HHS. ACTION: Notice. ----------------------------------------------------------------------- SUMMARY: 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 rare pediatric disease product applications that meet certain criteria. FDA has determined that CHOLBAM (cholic acid), manufactured by Asklepion Pharmaceuticals, LLC, meets the criteria for a priority review voucher. FOR FURTHER INFORMATION CONTACT: Larry Bauer, Rare Diseases Program, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave. Silver Spring, MD 20993-0002, 301-796-4842, FAX: 301-796-9858, email: [email protected]. SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. Under section 529 of the FD&C Act (21 U.S.C. 360ff), added by FDASIA, FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. FDA has determined that CHOLBAM (cholic acid), manufactured by Asklepion Pharmaceuticals, LLC, meets the criteria for a priority review voucher. CHOLBAM (cholic acid) is a bile acid indicated for the treatment of bile acid synthesis disorders due to single enzyme defects and as adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease or steatorrhea or complications from decreased fat soluble vitamin absorption. Bile acid synthesis disorders is a group of rare congenital disorders caused by the absence or malfunction of an enzyme involved in an important metabolic pathway, leading to a failure to produce normal bile acids. 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 http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further information about CHOLBAM (cholic acid), go to the [email protected] Web site at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm. Dated: April 2, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015-08016 Filed 4-6-15; 8:45 am] BILLING CODE CODE 4164-01-P
![18628 Federal Register / Vol. 80, No. 66 / Tuesday, April 7, 2015 / Notices
Regency, 1209 L Street, Sacramento, OHS will summarize oral testimony CHOLBAM (cholic acid), manufactured
California 95814 and comments from each Consultation by Asklepion Pharmaceuticals, LLC,
• July 30, 2015—Oklahoma Indian Head Session in the report without meets the criteria for a priority review
Start Coalition Conference, attribution, along with topics of concern voucher. CHOLBAM (cholic acid) is a
DoubleTree at Warren Place, 6110 and recommendations. OHS has sent bile acid indicated for the treatment of
South Yale Avenue, Tulsa, Oklahoma hotel and logistical information for the bile acid synthesis disorders due to
74136 California, Oklahoma, and Montana single enzyme defects and as adjunctive
• August 17, 2015—Northwest Indian Consultation Sessions to tribal leaders treatment of peroxisomal disorders,
Head Start Association Conference, via email and posted information on the including Zellweger spectrum disorders
Holiday Inn Grand Montana, 5500 Early Childhood Learning and in patients who exhibit manifestations
Midland Road, Billings, Montana Knowledge Center Web site at http:// of liver disease or steatorrhea or
59101 eclkc.ohs.acf.hhs.gov/hslc/hs/calendar/ complications from decreased fat
tc2015. soluble vitamin absorption. Bile acid
FOR FURTHER INFORMATION CONTACT: synthesis disorders is a group of rare
Robert Bialas, Regional Program Dated: March 26, 2015.
congenital disorders caused by the
Manager, Region XI, Office of Head Ann Linehan,
absence or malfunction of an enzyme
Start, email Robert.Bialas@acf.hhs.gov Acting Director, Office of Head Start. involved in an important metabolic
or phone (202) 205–9497. Additional [FR Doc. 2015–07958 Filed 4–6–15; 8:45 am] pathway, leading to a failure to produce
information and online meeting BILLING CODE CODE 4184–40–P normal bile acids.
registration is available at http://eclkc. For further information about the Rare
ohs.acf.hhs.gov/hslc/hs/calendar/ Pediatric Disease Priority Review
tc2015. DEPARTMENT OF HEALTH AND Voucher Program and for a link to the
HUMAN SERVICES full text of section 529 of the FD&C Act,
SUPPLEMENTARY INFORMATION: The
Department of Health and Human go to http://www.fda.gov/ForIndustry/
Food and Drug Administration DevelopingProductsforRare
Services (HHS) announces OHS Tribal
Consultations for leaders of Tribal [Docket No. FDA–2014–N–0229] DiseasesConditions/RarePediatric
Governments operating Head Start and DiseasePriorityVoucherProgram/
Issuance of Priority Review Voucher; default.htm.
Early Head Start programs. Rare Pediatric Disease Product For further information about
The agenda for the scheduled OHS
AGENCY: Food and Drug Administration, CHOLBAM (cholic acid), go to the
Tribal Consultations in Sacramento,
HHS. Drugs@FDA Web site at http://
California, Tulsa, Oklahoma, and www.accessdata.fda.gov/scripts/cder/
Billings, Montana, will be organized ACTION: Notice. drugsatfda/index.cfm.
around the statutory purposes of Head
Start Tribal Consultations related to SUMMARY: The Food and Drug Dated: April 2, 2015.
meeting the needs of American Indian/ Administration (FDA) is announcing the Leslie Kux,
Alaska Native children and families, issuance of a priority review voucher to Associate Commissioner for Policy.
taking into consideration funding the sponsor of a rare pediatric disease [FR Doc. 2015–08016 Filed 4–6–15; 8:45 am]
allocations, distribution formulas, and product application. The Federal Food, BILLING CODE CODE 4164–01–P
other issues affecting the delivery of Drug, and Cosmetic Act (the FD&C Act),
Head Start services in their geographic as amended by the Food and Drug
locations. In addition, OHS will share Administration Safety and Innovation DEPARTMENT OF HEALTH AND
actions taken and in progress to address Act (FDASIA), authorizes FDA to award HUMAN SERVICES
the issues and concerns raised in 2014 priority review vouchers to sponsors of
OHS Tribal Consultations. rare pediatric disease product Food and Drug Administration
The Consultation Sessions will be applications that meet certain criteria. [Docket No. FDA–2014–N–0229]
conducted with elected or appointed FDA has determined that CHOLBAM
leaders of Tribal Governments and their (cholic acid), manufactured by Issuance of Priority Review Voucher;
designated representatives [42 U.S.C. Asklepion Pharmaceuticals, LLC, meets Rare Pediatric Disease Product
9835, Section 640(l)(4)(A)]. Designees the criteria for a priority review
voucher. AGENCY: Food and Drug Administration,
must have a letter from the Tribal HHS.
Government authorizing them to FOR FURTHER INFORMATION CONTACT:
ACTION: Notice.
represent the tribe prior to the Larry Bauer, Rare Diseases Program,
Consultation Sessions. Other Center for Drug Evaluation and SUMMARY: The Food and Drug
representatives of tribal organizations Research, Food and Drug Administration (FDA) is announcing the
and Native nonprofit organizations are Administration, 10903 New Hampshire issuance of a priority review voucher to
welcome to attend as observers. Ave. Silver Spring, MD 20993–0002, the sponsor of a rare pediatric disease
A detailed report of the Consultation 301–796–4842, FAX: 301–796–9858, product application. The Federal Food,
Sessions will be prepared and made email: larry.bauer@fda.hhs.gov. Drug, and Cosmetic Act (the FD&C Act),
available within 45 days of the SUPPLEMENTARY INFORMATION: FDA is as amended by the Food and Drug
Consultation Sessions to all Tribal announcing the issuance of a priority Administration Safety and Innovation
mstockstill on DSK4VPTVN1PROD with NOTICES
Governments receiving funds for Head review voucher to the sponsor of a rare Act (FDASIA), authorizes FDA to award
Start and Early Head Start programs. pediatric disease product application. priority review vouchers to sponsors of
Tribes wishing to submit written Under section 529 of the FD&C Act (21 rare pediatric disease product
testimony for the report should send U.S.C. 360ff), added by FDASIA, FDA applications that meet certain criteria.
testimony to Robert Bialas at will award priority review vouchers to FDA has determined that UNITUXIN
Robert.Bialas@acf.hhs.gov either prior sponsors of rare pediatric disease (dinutuximab), manufactured by United
to the Consultation Sessions or within product applications that meet certain Therapeutics Corporation, meets the
30 days after the meeting. criteria. FDA has determined that criteria for a priority review voucher.
VerDate Sep<11>2014 17:46 Apr 06, 2015 Jkt 235001 PO 00000 Frm 00043 Fmt 4703 Sfmt 4703 E:\FR\FM\07APN1.SGM 07APN1](https://thefederalregister.org/doc/80_FR_18694/page/1.svg)
Document Created: 2018-02-21 10:05:37
Document Modified: 2018-02-21 10:05:37
| 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. | |
| Contact | Larry Bauer, Rare Diseases Program, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave. Silver Spring, MD 20993-0002, 301-796-4842, FAX: 301-796-9858, email: [email protected] | |
| FR Citation | 80 FR 18628 |
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