CENTER FOR DRUG EVALUATION AND
`RESEARCH
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`APPLICATION NUMBER:
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`211192Orig1s000
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`ADMINISTRATIVE and CORRESPONDENCE
`DOCUMENTS
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`

`

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`DEPARTMENT OF HEALTH AND HUMAN SERVICES
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`IND 119341
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`Food and Drug Administration
`Silver Spring MD 20993
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`MEETING MINUTES
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`Agios Pharmaceuticals, Inc.
`Attention: Shane A. McGann, PharmD, RPh
`Manager, Regulatory Affairs
`88 Sidney Street
`Cambridge, MA 02139
`
`
`Dear Dr. McGann:
`
`Please refer to your Investigational New Drug Application (IND) submitted under section 505(i)
`of the Federal Food, Drug, and Cosmetic Act for AG-120.
`
`We also refer to the meeting between representatives of your firm and the FDA on May 25,
`2016. The purpose of the meeting was to obtain guidance on the
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`Sincerely,
`
`{See appended electronic signature page}
`
`Donna Przepiorka, MD, PhD
`Acting Clinical Team Leader
`Division of Hematology Products
`Office of Hematology and Oncology Products
`Center for Drug Evaluation and Research
`
` A
`
`
`
`Enclosure:
`Meeting Minutes
`
` copy of the official minutes of the meeting is enclosed for your information. Please notify us
`of any significant differences in understanding regarding the meeting outcomes.
`
`If you have any questions, call Laura Wall, Regulatory Project Manager at (301) 796-2237.
`
`
`Reference ID: 3943191
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`(b) (4)
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`

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`FOOD AND DRUG ADMINISTRATION
`CENTER FOR DRUG EVALUATION AND RESEARCH
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`Meeting Type:
`Meeting Category:
`
`Meeting Date and Time: May 25, 2016 from 2:00 PM to 3:00 PM (ET)
`Meeting Location:
`White Oak Building 22, Conference Room: 1313
`
`Application Number:
`Product Name:
`Proposed Indication:
`
`
`MEMORANDUM OF MEETING MINUTES
`
`
`Type B
`Pre-Phase 3
`
`IND 119341
`AG-120
`Treatment of patients with acute myelogenous leukemia (AML)
`harboring an isocitrate dehydrogenase-1 (IDH1) mutation:
`• As a single agent for the treatment of adult patients with relapsed
`or refractory (R/R) AML
`
`Donna Przepiorka, MD, PhD, Acting Clinical Team Leader
`Laura Wall, MS, Regulatory Project Manager
`
`Sponsor/Applicant Name: Agios Pharmaceuticals, Inc.
`
`Meeting Chair:
`Meeting Recorder:
`
`FDA ATTENDEES
`Division of Hematology Products (DHP)
`Ann Farrell, MD, Division Director
`Albert Deisseroth, MD, PhD, Clinical Team Leader
`Donna Przepiorka, MD, PhD, Acting Clinical Team Leader
`Pat Dinndorf, MD, Clinical Reviewer
`Ashley Ward, MD, Clinical Reviewer,
`Laura Wall, MS, Regulatory Project Manager
`
`Office of Clinical Pharmacology, Division of Pharmacometrics
`Olanrewaju Okusanya, PharmD, MS, Clinical Pharmacology Reviewer
`
`Office of Biostatistics, Division of Biometrics V
`Lei Nie, PhD, Team Leader
`Kallappa Koti, PhD, Statistical Reviewer
`
`Division of Hematology Oncology Toxicology (DHOT)
`Christopher Sheth, PhD, Supervisory Pharmacologist/Toxicologist
`Matthew Thompson, PhD, MPH, Pharmacology/Toxicology Reviewer
`
`
`
`
`Reference ID: 3943191
`
`(b) (4)
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`

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`IND 119341
`Page 2
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`
`Office of Translational Sciences
`Sarah Dorff, PhD, Genomics Reviewer
`
`SPONSOR ATTENDEES
`Chris Bowden, MD, Agios, Chief Medical Officer
`Sam Agresta, MD, MPH & TM, MS CI & TR, Agios, Vice-President, Clinical Development
`Ann Cahill, PA, Agios, Senior Director, Clinical Development
`Eyal Attar, MD, Agios, Medical Director, Clinical Development
`Meredith Goldwasser, ScD, Agios, Senior Director, Head of Biometrics and Data Management
`Hua Liu, PhD, Agios, Associate Director of Biostatistics
`Jacqueline Cinicola, MS, Agios, Senior Director, Regulatory Affairs
`Shane McGann, PharmD, RPh, Agios, Manager, Regulatory Affairs
`Annie Estrella, MS, Agios, Director, Head of Medical Writing
`Katharine Yen, PhD, Agios, Director, Clinical Science
`Paul McInulty, Celgene, Executive Director, Global Regulatory Affairs
`Krishnan Viswanadhan, PharmD, MBA, Celgene, Global Project Leadership, Alliance Partner
`
`
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`1.0
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`BACKGROUND
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`Reference ID: 3943191
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`(b) (4)
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`(b) (4)
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`2 Page(s) has been Withheld in Full as b4 (CCI/TS) immediately
`following this page
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`IND 119341
`Page 5
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`3.0 OTHER IMPORTANT MEETING INFORMATION
`
`
`PREA REQUIREMENTS
`
`Under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), all applications for new
`active ingredients (which includes new salts and new fixed combinations), new indications, new
`dosage forms, new dosing regimens, or new routes of administration are required to contain an
`assessment of the safety and effectiveness of the product for the claimed indication(s) in
`pediatric patients unless this requirement is waived, deferred, or inapplicable.
`
`Because this drug product for this indication has an orphan drug designation, you are exempt
`from these requirements. Please include a statement that confirms this finding, along with a
`reference to this communication, as part of the pediatric section (1.9 for eCTD submissions) of
`your application. If there are any changes to your development plans that would cause your
`application to trigger PREA, your exempt status would change.
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`Reference ID: 3943191
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`(b) (4)
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`

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`IND 119341
`Page 6
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`DATA STANDARDS FOR STUDIES
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`Under section 745A(a) of the FD&C Act, electronic submissions “shall be submitted in such
`electronic format as specified by [FDA].” FDA has determined that study data contained in
`electronic submissions (i.e., NDAs, BLAs, ANDAs and INDs) must be in a format that the
`Agency can process, review, and archive. Currently, the Agency can process, review, and
`archive electronic submissions of clinical and nonclinical study data that use the standards
`specified in the Data Standards Catalog (Catalog) (See
`http://www.fda.gov/forindustry/datastandards/studydatastandards/default.htm).
`
`On December 17, 2014, FDA issued final guidance, Providing Electronic Submissions in
`Electronic Format--- Standardized Study Data
`(http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/
`UCM292334.pdf). This guidance describes the submission types, the standardized study data
`requirements, and when standardized study data will be required. Further, it describes the
`availability of implementation support in the form of a technical specifications document, Study
`Data Technical Conformance Guide (Conformance Guide) (See
`http://www.fda.gov/downloads/ForIndustry/DataStandards/StudyDataStandards/UCM384744.pd
`f), as well as email access to the eData Team (cder-edata@fda.hhs.gov) for specific questions
`related to study data standards. Standardized study data will be required in marketing
`application submissions for clinical and nonclinical studies that start on or after December 17,
`2016. Standardized study data will be required in commercial IND application submissions for
`clinical and nonclinical studies that start on or after December 17, 2017. CDER has produced a
`Study Data Standards Resources web page that provides specifications for sponsors regarding
`implementation and submission of clinical and nonclinical study data in a standardized
`format. This web page will be updated regularly to reflect CDER's growing experience in order
`to meet the needs of its reviewers.
`
`Although the submission of study data in conformance to the standards listed in the FDA Data
`Standards Catalog will not be required in studies that start before December 17, 2016, CDER
`strongly encourages IND sponsors to use the FDA supported data standards for the submission of
`IND applications and marketing applications. The implementation of data standards should
`occur as early as possible in the product development lifecycle, so that data standards are
`accounted for in the design, conduct, and analysis of clinical and nonclinical studies. For clinical
`and nonclinical studies, IND sponsors should include a plan (e.g., in the IND) describing the
`submission of standardized study data to FDA. This study data standardization plan (see the
`Conformance Guide) will assist FDA in identifying potential data standardization issues early in
`the development program.
`
`Additional information can be found at
`http://www.fda.gov/Drugs/DevelopmentApprovalProcess/FormsSubmissionRequirements/Electr
`onicSubmissions/ucm248635.htm.
`
`For general toxicology, supporting nonclinical toxicokinetic, and carcinogenicity studies,
`CDER encourages sponsors to use Standards for the Exchange of Nonclinical Data (SEND) and
`submit sample or test data sets before implementation becomes required. CDER will provide
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`Reference ID: 3943191
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`IND 119341
`Page 7
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`feedback to sponsors on the suitability of these test data sets. Information about submitting a test
`submission can be found here:
`http://www.fda.gov/Drugs/DevelopmentApprovalProcess/FormsSubmissionRequirements/Electr
`onicSubmissions/ucm174459.htm.
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`LABORATORY TEST UNITS FOR CLINICAL TRIALS
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`CDER strongly encourages IND sponsors to identify the laboratory test units that will be
`reported in clinical trials that support applications for investigational new drugs and product
`registration. Although Système International (SI) units may be the standard reporting
`mechanism globally, dual reporting of a reasonable subset of laboratory tests in U.S.
`conventional units and SI units might be necessary to minimize conversion needs during review.
`Identification of units to be used for laboratory tests in clinical trials and solicitation of input
`from the review divisions should occur as early as possible in the development process. For
`more information, please see the FDA website entitled, Study Data Standards Resources and the
`CDER/CBER Position on Use of SI Units for Lab Tests website found at
`http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/ucm372553.htm.
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`NEW PROTOCOLS AND CHANGES TO PROTOCOLS
`
`To ensure that the Division is aware of your continued drug development plans and to facilitate
`successful interactions with the Division, including provision of advice and timely responses to
`your questions, we request that the cover letter for all new phase 2 or phase 3 protocol
`submissions to your IND or changes to these protocols include the following information:
`
`
`1. Study phase
`2. Statement of whether the study is intended to support marketing and/or labeling changes
`3. Study objectives (e.g., dose finding)
`4. Population
`5. A brief description of the study design (e.g., placebo or active controlled)
`6. Specific concerns for which you anticipate the Division will have comments
`7. For changes to protocols only, also include the following information:
`• A brief summary of the substantive change(s) to the protocol (e.g., changes to
`endpoint measures, dose, and/or population)
`• Other significant changes
`• Proposed implementation date
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`ISSUES REQUIRING FURTHER DISCUSSION
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`We recommend you consider requesting a meeting to facilitate discussion of multiple and/or
`complex issues.
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`4.0
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`None
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`5.0
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`ACTION ITEMS
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`Reference ID: 3943191
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`

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`IND 119341
`Page 8
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`None
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`6.0
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`Attached are Agios’ responses to the Agency’s preliminary comments and the slides presented at
`the meeting. The responses were received via e-mail on May 23, 2016, and the slides were
`received via e-mail on May 24, 2016.
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`ATTACHMENTS AND HANDOUTS
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`
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`Reference ID: 3943191
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`15 Page(s) has been Withheld in Full as b4 (CCI/TS)
`immediately following this page
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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/
`----------------------------------------------------
`
`DONNA PRZEPIORKA
`06/08/2016
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`Reference ID: 3943191
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`