Document ID: FDA-2015-N-0001-0061
Agency: fda
Document Type: Notice
Title: New Methods To Predict the Immunogenicity of Therapeutic Coagulation Proteins; Public Workshop
Posted Date: 2015-07-02T04:00Z

[Federal Register Volume 80, Number 127 (Thursday, July 2, 2015)]
[Notices]
[Page 38210]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-16365]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-0001]

New Methods To Predict the Immunogenicity of Therapeutic 
Coagulation Proteins; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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    The Food and Drug Administration (FDA) is announcing a public 
workshop entitled: ``New Methods to Predict the Immunogenicity of 
Therapeutic Coagulation Proteins''. The purpose of the public workshop 
is to discuss recent scientific progress in identifying the genetic 
determinants for an unwanted immune response to therapeutic coagulation 
proteins (immunogenicity), and to identify and discuss potential new 
methods to predict such immunogenicity. Immunogenicity results in the 
development of antibodies that target the therapeutic protein and can 
affect the safety and efficacy of the biological product. The workshop 
has been planned in partnership with the National Heart, Lung and Blood 
Institute, National Institutes of Health (NIH), the National Hemophilia 
Foundation, and the Plasma Protein Therapeutics Association. The 
workshop will include presentations and panel discussions by experts 
from academic institutions, industry, and government Agencies.
    Date and Time: The public workshop will be held on September 17, 
2015, from 8:30 a.m. to 5 p.m. and on September 18, 2015, from 8:30 
a.m. to 12 p.m.
    Location: The public workshop will be held at the Ruth Kirschstein 
Auditorium, Natcher Conference Center, Bldg. 45, National Institutes of 
Health Campus, 9000 Rockville Pike, Bethesda, MD 20892. The entrance 
for the public workshop participants (non-NIH employees) is through the 
NIH Gateway Center located adjacent to the Medical Center Metro, where 
routine security check procedures will be performed. Please visit the 
following Web site for location, parking, security, and travel 
information: http://www.nih.gov/about/visitor/index.htm. Please visit 
the following Web site for information on the Natcher Conference 
Center: http://www.genome.gov/11007522.
    Contact Person: Freddy Barnes, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993, 240-402-6943, William.Barnes@fda.hhs.gov. For 
questions email: CBERPublicEvents@fda.hhs.gov (Subject line: FDA 
MPICPDT Workshop).
    Registration: Please visit the following Web site to register for 
the workshop by August 27, 2015: http://methodspredictimmunogenicity.eventbrite.com. There is no registration 
fee for the public workshop. Early registration is recommended because 
seating is limited. Registration on the day of the public workshop will 
be provided on a space available basis beginning at 8:15 a.m.
    If you need special accommodations due to a disability, please 
contact Freddy Barnes (see Contact Person) at least 7 days in advance.
    Supplementary Information: The development of unwanted immune 
responses to therapeutic coagulation protein products may affect both 
product efficacy and patient safety. In the case of replacement 
coagulation protein therapies, the inhibitory anti-drug antibodies also 
interact with the endogenous protein and may result in serious adverse 
events in patients. Both product and patient specific factors may 
affect the immunogenicity of therapeutic coagulation protein products. 
There are currently several initiatives underway to assess the genetic 
basis for developing unwanted immune responses to coagulation protein 
products in individuals with hemophilia, which will result in the 
accumulation of large data sets over the next few years. The workshop 
aims to address what patients, healthcare professionals and regulators 
may do with this information to improve patient outcomes.
    In addition, an unprecedented number of new engineered recombinant 
coagulation proteins are in development. This workshop will discuss the 
state-of-the art with respect to leveraging scientific progress to 
predict the immunogenicity of protein amino acid sequences that do not 
exist in nature, and whether there is a need for novel strategies in 
the design and conduct of clinical trials for these products.
    The first day of the workshop will include presentations and panel 
discussions on the following topics: (1) Overview of the current 
understanding of genetic factors that affect immunogenicity of 
therapeutic coagulation proteins; (2) recent advances in immunology 
relevant to immunogenicity; (3) emerging computational, in vitro and ex 
vivo tools to predict the immunogenicity of therapeutic coagulation 
proteins and how these tools may be evaluated in a clinical setting; 
and (4) initiatives to determine the genetic factors that affect 
immunogenicity of coagulation protein products in individuals with 
hemophilia and strategies to optimize the outcome data.
    The second day of the workshop will include presentations and panel 
discussions on the following topics: (1) Challenges related to the 
development of novel recombinant coagulation protein products; (2) a 
round-table discussion and question and answer session; and (3) 
workshop summary.
    Transcripts: Please be advised that as soon as possible after a 
transcript of this public workshop will be available, it will be 
accessible at: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm438035.htm. Transcripts of the public 
workshop may also be requested in writing from the Division of Freedom 
of Information (ELEM-1029), Food and Drug Administration, 12420 
Parklawn Dr., Rockville, MD 20857.

    Dated: June 29, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-16365 Filed 7-1-15; 8:45 am]
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