Document ID: FDA-2014-N-0001-0045
Agency: fda
Document Type: Notice
Title: Immune Responses to Enzyme Replacement Therapies: Role of
Immune Tolerance Induction; Public Workshop
Posted Date: 2014-05-13T04:00Z

[Federal Register Volume 79, Number 92 (Tuesday, May 13, 2014)]
[Notices]
[Page 27313]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-10933]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0001]

Immune Responses to Enzyme Replacement Therapies: Role of Immune 
Tolerance Induction; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration's (FDA) Center for Drug 
Evaluation and Research, in cosponsorship with the National 
Organization for Rare Disorders (NORD), is announcing a 1-day public 
workshop entitled ``Immune Responses to Enzyme Replacement Therapies: 
Role of Immune Tolerance Induction.'' Partners and stakeholders 
planning the workshop also include representatives from academia, 
industry, and patients. The purpose of this workshop is to provide a 
forum to discuss the role of immune tolerance induction in patients 
receiving replacement biological products.

DATES: The public workshop will be held on June 9, 2014, from 8 a.m. to 
5 p.m.

ADDRESSES: The public workshop will be held at FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room 
(Rm. 1503), Silver Spring, MD 20993-0002. Entrance for the public 
workshop participants (non-FDA employees) is through Bldg. 1 where 
routine security check procedures will be performed. For parking and 
security information, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: Maureen Dewey, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-0845, FAX: 301-
796-9905, Maureen.Dewey@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: FDA's Center for Drug Evaluation and 
Research, in co-sponsorship with NORD, is announcing a 1-day public 
workshop entitled ``Immune Responses to Enzyme Replacement Therapies: 
Role of Immune Tolerance Induction.'' The cosponsored workshop will 
facilitate an ongoing dialogue among relevant parties on issues related 
to the role of immune tolerance induction in enzyme replacement 
therapies. The workshop will discuss the impact of anti-drug and 
neutralizing antibodies on efficacy and safety of enzyme replacement 
therapies intended to treat patients with lysosomal storage diseases 
and the risks and benefits of implementing prophylactic immune 
tolerance regimens to preclude generation of these antibodies. 
Stakeholders, including patients and patient organizations, industry 
sponsors, academia, and FDA, will discuss challenging issues related to 
immune tolerance induction in enzyme replacement therapies.
    Registration: There is no fee to attend the public workshop, but 
advanced online registration is requested. Space is limited, and 
registration will be on a first-come, first-served basis. To register 
online, please visit https://events.rarediseases.org/?page_id=4ⅇ=13. 
Onsite registration the day of the workshop will be available, but 
advanced registration is preferred.
    If you need special accommodations due to a disability, please 
contact Maureen Dewey (see FOR FURTHER INFORMATION CONTACT) at least 7 
days in advance.
    Transcripts: Transcripts of the workshop will be available for 
review at the Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, and 
at http://www.regulations.gov approximately 30 days after the workshop. 
A transcript will also be available in either hardcopy or on CD-ROM, 
after submission of a Freedom of Information request. Send written 
requests to the Division of Freedom of Information (ELEM-1029), Food 
and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, 
MD 20857. Send faxed requests to 301-827-9267.

    Dated: May 7, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-10933 Filed 5-12-14; 8:45 am]
BILLING CODE 4160-01-P