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` NDA 021825/S-008
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`SUPPLEMENT APPROVAL
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` Chiesi USA, Inc
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` Attention: Matt D. Medlin, PhD, RAC
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` Head of US Regulatory Affairs, Global Rare Diseases
` 175 Regency Woods PIace, Suite 600
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` Cary, NC 27518
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`Dear Dr. Medlin:
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`Please refer to your supplemental new drug applications (sNDA) dated and received
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`June 30, 2020, and your amendments, submitted under section 505(b)of the Federal
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`Food, Drug, and Cosmetic Act (FDCA) for Ferriprox (deferiprone) tablets.
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`This Prior Approval supplemental new drug application provides the new indication for
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`the treatment of patients with transfusional iron overload due to sickle cell disease or
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`other anemias.
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`APPROVAL & LABELING
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`We have completed our review of this application, as amended. It is approved, effective
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`on the date of this letter, for use as recommended in the enclosed agreed-upon
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`labeling.
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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
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`content of labeling [21 CFR 314.50(l)] in structured product labeling (SPL) format using
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`the FDA automated drug registration and listing system (eLIST), as described at
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`FDA.gov.1 Content of labeling must be identical to the enclosed labeling (text for the
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`Prescribing Information and Medication Guide), with the addition of any labeling
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`changes in pending “Changes Being Effected” (CBE) supplements, as well as annual
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`reportable changes not included in the enclosed labeling.
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`Information on submitting SPL files using eList may be found in the guidance for
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`industry SPL Standard for Content of Labeling Technical Qs and As.2
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` 1 http://www.fda.gov/ForIndustry/DataStandards/StructuredProductLabeling/default.htm
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` 2 We update guidances periodically. For the most recent version of a guidance, check the FDA Guidance
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` Documents Database https://www.fda.gov/RegulatoryInformation/Guidances/default.htm.
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`Reference ID: 4788713
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` NDA 021825/S-008
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` Page 2
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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 include labeling
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` changes for this NDA, including CBE supplements for which FDA has not yet issued an
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` action letter, with the content of labeling [21 CFR 314.50(l)(1)(i)] in Microsoft Word
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` format, that includes the changes approved in this supplemental application, as well as
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` annual reportable changes. To facilitate review of your submission(s), provide a
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` highlighted or marked-up copy that shows all changes, as well as a clean Microsoft
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`Word version. The marked-up copy should provide appropriate annotations, including
`supplement number(s) and annual report date(s).
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` REQUIRED PEDIATRIC ASSESSMENTS
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`Under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), all applications for
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`new active ingredients (which includes new salts and new fixed combinations), new
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`indications, new dosage forms, new dosing regimens, or new routes of administration
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`are required to contain an assessment of the safety and effectiveness of the product for
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`the claimed indication in pediatric patients unless this requirement is waived, deferred,
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`or inapplicable.
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`Because this drug product for this indication has an orphan drug designation, you are
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`exempt from this requirement.
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`SUBPART H FULFILLED
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`We approved this NDA under the regulations at 21 CFR 314 Subpart H for accelerated
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`approval of new drugs for serious or life-threatening illnesses. Approval of this
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`supplement fulfills your commitments made under 21 CFR 314.510.
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`FULFILLMENT OF POSTMARKETING REQUIREMENT(S)/COMMITMENT(S)
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`We have received your submission dated June 30, 2020, containing the final reports for
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`the following postmarketing requirements listed in the October 14, 2011, approval letter.
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`1828-1
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`Conduct a trial to determine the efficacy and safety of the use of
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`deferiprone to treat iron overload in patients with sickle cell disease and
`transfusional hemosiderosis who have not been adequately treated with
`available chelating agents. Submit the protocol for review and
`concurrence prior to commencing. The trial will enroll a sufficient number
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`of patients with sickle cell disease as described above, to provide
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`sufficient evidence to assess the efficacy and safety in the sickle cell
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`disease population described. The trial may enroll patients with other
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`conditions who have developed transfusional iron overload. The trial will
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`stratify for hematologic diagnosis for the randomization. The primary and
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`U.S. Food and Drug Administration
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`Silver Spring, MD 20993
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`www.fda.gov
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`Reference ID: 4788713
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` NDA 021825/S-008
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` Page 3
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`secondary endpoints will measure changes in cardiac iron concentration
`and liver iron concentration
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` Final Protocol Submission: 02/2012
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` 01/2016
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` Trial Completion:
` Final Report Submission: 07/2016
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`We have reviewed your submission and conclude that the above
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`requirements/commitments were fulfilled.
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`This completes all of your postmarketing requirements and postmarketing commitments
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`acknowledged in our October 14, 2011, letter.
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`PROMOTIONAL MATERIALS
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`You may request advisory comments on proposed introductory advertising and
`promotional labeling. For information about submitting promotional materials, see the
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`final guidance for industry Providing Regulatory Submissions in Electronic and Non-
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`Electronic Format-Promotional Labeling and Advertising Materials for Human
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`Prescription Drugs.3
`You must submit final promotional materials and Prescribing Information, accompanied
`by a Form FDA 2253, at the time of initial dissemination or publication
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`[21 CFR 314.81(b)(3)(i)]. Form FDA 2253 is available at FDA.gov.4 Information and
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`Instructions for completing the form can be found at FDA.gov.5
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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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` 3 For the most recent version of a guidance, check the FDA guidance web page at
` https://www.fda.gov/media/128163/download.
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` 4 http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Forms/UCM083570.pdf
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` 5 http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Forms/UCM375154.pdf
`U.S. Food and Drug Administration
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`Silver Spring, MD 20993
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`www.fda.gov
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`Reference ID: 4788713
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`NDA 021825/S-008
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`Page 4
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`If you have any questions, call May Zuwannin, Regulatory Project Manager, at
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`301-796-7775.
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`Sincerely,
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`{See appended electronic signature page}
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`Ann Farrell, MD
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`Director
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`Division of Nonmalignant Hematology
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`Office of Cardiology, Hematology, Endocrinology,
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`and Nephrology
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`Center for Drug Evaluation and Research
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`ENCLOSURES:
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`• Content of Labeling
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`o Prescribing Information
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`o Medication Guide
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`U.S. Food and Drug Administration
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`Silver Spring, MD 20993
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`www.fda.gov
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`Reference ID: 4788713
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`Signature Page 1 of 1
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`This is a representation of an electronic record that was signed
`electronically. Following this are manifestations of any and all
`electronic signatures for this electronic record.
`--------------------------------------------------------------------------------------------
`/s/
`------------------------------------------------------------
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`ANN T FARRELL
`04/30/2021 02:44:41 PM
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`Reference ID: 4788713
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