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`DEPARTMENT OF HEALTH AND HUMAN SERVICES
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` NDA 203284/S-01
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`Food and Drug Administration
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` Silver Spring MD 20993
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`SUPPLEMENT APPROVAL
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` Hyperion Therapeutics
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` Attention: Klara A. Dickinson-Eason
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` SVP, Chief Regulatory Officer and Corporate Quality
` 2000 Sierra Point Parkway, Suite 400
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` Brisbane, CA 94005
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`Dear Ms. Dickinson-Eason:
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`Please refer to your Supplemental New Drug Application (sNDA) dated and received
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`May 6, 2014, submitted under section 505(b) of the Federal Food, Drug, and Cosmetic Act
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`(FDCA) for Ravicti (glycerol phenylbutyrate) Oral Liquid.
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`This “Prior Approval” supplemental new drug application provides for the following revised
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`langue in Section 8.3 Nursing Mothers:
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`Breastfeeding is not recommended with maternal use of RAVICTI. It is not known
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`whether RAVICTI or its metabolites are present in breast milk. Because many drugs are
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`present in breast milk and because of the potential for tumorigencity of glycerol
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`phenylbutyrate identified in animal studies, as well as the potential for serious adverse
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`reactions in nursing infants from RAVICTI, a decision should be made whether to
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`discontinue nursing or to discontinue the drug, taking into consideration the importance
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`of the drug to the health of the mother [see Use in Specific Populations (8.4) and
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`Nonclinical Toxicology (13.1)].
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`APPROVAL & LABELING
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`We have completed our review of this supplemental application. It is approved, effective on the
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`date of this letter, for use as recommended in the enclosed, agreed-upon labeling text.
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`CONTENT OF LABELING
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`As soon as possible, but no later than 14 days from the date of this letter, submit the content of
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`labeling [21 CFR 314.50(l)] in structured product labeling (SPL) format using the FDA
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`automated drug registration and listing system (eLIST), as described at
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`http://www.fda.gov/ForIndustry/DataStandards/StructuredProductLabeling/default.htm. Content
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`of labeling must be identical to the enclosed labeling (text for the package insert and Medication
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`Guide), with the addition of any labeling changes in pending “Changes Being Effected” (CBE)
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`supplements, as well as annual reportable changes not included in the enclosed labeling.
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`Reference ID: 3530007
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` NDA 203284/S-01
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` Page 2
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` Information on submitting SPL files using eList may be found in the guidance for industry titled
` “SPL Standard for Content of Labeling Technical Qs and As at
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`http://www.fda.gov/downloads/DrugsGuidanceComplianceRegulatoryInformation/Guidances/U
`CM072392.pdf
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` The SPL will be accessible from publicly available labeling repositories.
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`Also within 14 days, amend all pending supplemental applications that includes labeling changes
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`for this NDA, including CBE supplements for which FDA has not yet issued an action letter,
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`with the content of labeling [21 CFR 314.50(l)(1)(i)] in MS Word format, that includes the
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`changes approved in this supplemental application, as well as annual reportable changes and
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`annotate each change. To facilitate review of your submission, provide a highlighted or marked-
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`up copy that shows all changes, as well as a clean Microsoft Word version. The marked-up copy
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`should provide appropriate annotations, including supplement number(s) and annual report
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`date(s).
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`REPORTING REQUIREMENTS
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`We remind you that you must comply with reporting requirements for an approved NDA
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`(21 CFR 314.80 and 314.81).
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`If you have any questions, call Jessica Benjamin, Regulatory Project Manager, at (301) 796­
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`3924.
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`Sincerely,
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`{See appended electronic signature page}
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`Joyce Korvick, M.D., M.P.H.
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`Deputy Director for Safety
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`Division of Gastroenterology and Inborn Errors
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`Products
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`Office of Drug Evaluation III
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`Center for Drug Evaluation and Research
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`ENCLOSURE:
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`Content of Labeling
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`Reference ID: 3530007
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`---------------------------------------------------------------------------------------------------------
`This is a representation of an electronic record that was signed
`electronically and this page is the manifestation of the electronic
`signature.
`---------------------------------------------------------------------------------------------------------
`/s/
`----------------------------------------------------
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`JOYCE A KORVICK
`06/24/2014
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`Reference ID: 3530007
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