Notice2024-19334

Issuance of Priority Review Voucher; Rare Pediatric Disease Product; LIVMARLI (maralixibat)

Primary source

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Published
August 28, 2024

Issuing agencies

Health and Human Services DepartmentFood and Drug Administration

Abstract

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 (FD&C Act) 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 LIVMARLI (maralixibat), approved on September 29, 2021, manufactured by Mirum Pharmaceuticals, Inc., meets the criteria for a priority review voucher.

Full Text

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<title>Federal Register, Volume 89 Issue 167 (Wednesday, August 28, 2024)</title>
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[Federal Register Volume 89, Number 167 (Wednesday, August 28, 2024)]
[Notices]
[Page 68909]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-19334]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-N-0026]


Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product; LIVMARLI (maralixibat)

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 the sponsor of a rare 
pediatric disease product application. The Federal Food, Drug, and 
Cosmetic Act (FD&C Act) 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 LIVMARLI (maralixibat), approved on September 29, 2021, 
manufactured by Mirum Pharmaceuticals, Inc., meets the criteria for a 
priority review voucher.

FOR FURTHER INFORMATION CONTACT: Cathryn Lee, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-1394, 
<a href="/cdn-cgi/l/email-protection#97d4f6e3ffe5eef9b9dbf2f2d7f1f3f6b9ffffe4b9f0f8e1"><span class="__cf_email__" data-cfemail="c083a1b4a8b2b9aeee8ca5a580a6a4a1eea8a8b3eea7afb6">[email&#160;protected]</span></a>.

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), FDA will award priority review vouchers to sponsors of approved 
rare pediatric disease product applications that meet certain criteria. 
FDA has determined that LIVMARLI (maralixibat), manufactured by Mirum 
Pharmaceuticals, Inc., meets the criteria for a priority review 
voucher. LIVMARLI (maralixibat) oral solution is indicated for the 
treatment of cholestatic pruritus in patients with Alagille syndrome 
(ALGS) 1 year of age and older.
    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 <a href="https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm">https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm</a>. For further 
information about LIVMARLI (maralixibat), go to the ``Drugs@FDA'' 
website at <a href="https://www.accessdata.fda.gov/scripts/cder/daf/">https://www.accessdata.fda.gov/scripts/cder/daf/</a>.

    Dated: August 23, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-19334 Filed 8-27-24; 8:45 am]
BILLING CODE 4164-01-P


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Indexed from Federal Register on August 28, 2024.

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