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2019-08-08
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Fabry Disease: Developing Drugs for Treatment; Draft Guidance for Industry; Availability
Notices
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United States
Department of Health and Human Services
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United States Government Agency or Subagency
United States
Food and Drug Administration
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United States Government Agency or Subagency
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Fabry Disease: Developing Drugs for Treatment.'' This draft guidance describes the Agency's current recommendations regarding eligibility criteria, trial design considerations, and efficacy endpoints to be used in clinical development programs of investigational drugs to treat Fabry disease. Through this draft guidance, the Agency provides clear and specific guidance to foster greater efficiency in drug development in this rare disease with the goal of enhancing clinical trial data quality and supporting the development of treatments for Fabry disease.
84 FR 38994
https://www.govinfo.gov/app/details/FR-2019-08-08/2019-16994
2019-16994
fr08au19-69
4164-01-P
Docket No. FDA-2019-D-2973
https://www.govinfo.gov/app/details/FR-2019-08-08/2019-16994
https://www.govinfo.gov/content/pkg/FR-2019-08-08/html/2019-16994.htm
https://www.govinfo.gov/content/pkg/FR-2019-08-08/pdf/2019-16994.pdf
3 p.
38994
38996
84 FR 38994
Fabry Disease: Developing Drugs for Treatment; Draft Guidance for Industry; Availability; Federal Register Vol. 84, Issue
NOTICE
2019-16994
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
2019-11-06
Docket No. FDA-2019-D-2973
4164-01-P
2019-16994
Notice of availability.
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Fabry Disease: Developing Drugs for Treatment.'' This draft guidance describes the Agency's current recommendations regarding eligibility criteria, trial design considerations, and efficacy endpoints to be used in clinical development programs of investigational drugs to treat Fabry disease. Through this draft guidance, the Agency provides clear and specific guidance to foster greater efficiency in drug development in this rare disease with the goal of enhancing clinical trial data quality and supporting the development of treatments for Fabry disease.
Submit either electronic or written comments on the draft guidance by November 6, 2019 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
Jeannie Roule, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 5332, Silver Spring, MD 20993-0002, 301- 796-3993; or Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.
Guidance:
Fabry Disease: Developing Drugs for Treatment
,
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs
https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics
https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf
https://www.regulations.gov
Federal Register
Vol. 84, no. 153
Office of the Federal Register, National Archives and Records Administration
2019-08-08
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335 p.
Table of Contents:
AE 2.7:
GS 4.107:
AE 2.106:
KF70.A2
https://www.govinfo.gov/app/details/FR-2019-08-08
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https://www.govinfo.gov/app/details/FR-2019-08-08
https://www.govinfo.gov/content/pkg/FR-2019-08-08/pdf/FR-2019-08-08.pdf
https://www.govinfo.gov/content/pkg/FR-2019-08-08/xml/FR-2019-08-08.xml
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2019-08-08
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FR-2019-08-08
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FR-2019-08-08
84
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