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Federal Register / Vol. 86, No. 233 / Wednesday, December 8, 2021 / Notices heading of this document, into the Search box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 2404027500.
You may submit comments on any guidance at any time see 21 CFR
10.115g5.
Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 20993
0002. Send one self-addressed adhesive label to assist that office in processing your requests. See the SUPPLEMENTARY
INFORMATION section for electronic access to the draft guidance document.
FOR FURTHER INFORMATION CONTACT:
Hobart Rogers, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Bldg. 51, Rm 3114, Silver Spring, MD 209030002, 301
7962213.
SUPPLEMENTARY INFORMATION:

jspears on DSK121TN23PROD with NOTICES1

I. Background FDA is announcing the availability of a draft guidance for sponsorinvestigators entitled IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or LifeThreatening Diseases: Clinical Recommendations. FDA is publishing this draft guidance to provide sponsorinvestigators hereafter referred to as sponsors who are interested in developing individualized ASO drug products for a rapidly progressive SDLT
genetic disease caused by a unique genetic variant or variants, with clinical recommendations for submission of INDs. These recommendations specifically address the following clinical considerations: Ethical and human subject protection, diagnostic and genetic, dosing, administration, safety, and assessment of clinical response to treatment.
This draft guidance describes clinical considerations and, when applicable, important information to include in IND
submissions for such ASO drug products for a small number of participants typically one to two with SDTL diseases. In general, ASO drug products referred to in this draft guidance belong to a well-characterized chemical class and for which there is considerable nonclinical and clinical experience that is publicly available or to which the sponsor has a right of reference. The draft guidance discusses considerations and information to
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submit in an IND regarding: 1
Confirmation of the participants genetic diagnosis and genetic variants targeted by the ASO drug product, 2 the requirements and procedures for informed consent of the participant, 3
appropriate and safe dosing and administration procedures that are detailed and supported by relevant nonclinical evidence, 4 the nature and schedule of the specific safety assessments adverse events and laboratory testing to be conducted, and 5 methods for continuous clinical monitoring e.g., via clinical outcome assessments, pharmacodynamic biomarkers of the participant to evaluate and document their clinical responses and to allow for an informed benefit-risk determination. This draft guidance is expected to facilitate the preparation of adequate and complete IND submissions for investigational ASO drug products for participants with SDLT diseases targeted by the specified ASO drug product.
This draft guidance represents one guidance in a series of guidances that FDA intends to publish to advise and help sponsors planning to use individualized ASO drug products for SDLT diseases caused by unique genetic variants and for whom there are no alternative therapies available to treat their disease.
This draft guidance is being issued consistent with FDAs good guidance practices regulation 21 CFR 10.115.
The draft guidance, when finalized, will represent the current thinking of FDA
on IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations. It does not establish any rights for any person and is not binding on FDA or the public.
You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
While this guidance contains no new collection of information, it does refer to previously approved FDA collections of information. Therefore, clearance by the Office of Management and Budget OMB under the Paperwork Reduction Act of 1995 PRA 44 U.S.C. 3501
3521 is not required for this guidance.
The previously approved collections of information are subject to review by OMB under the PRA. The collections of information in 21 CFR part 312 have been approved under OMB control number 09100014. The collections of information in 21 CFR parts 50 and 56
for obtaining informed consent for prospective patients have been
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approved under OMB control number 09100130.
III. Electronic Access Persons with access to the internet may obtain the draft guidance at either https www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https
www.regulations.gov.
Dated: December 1, 2021.
Lauren K. Roth, Associate Commissioner for Policy.
FR Doc. 202126453 Filed 12721; 8:45 am BILLING CODE 416401P

DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration Docket No. FDA2021D1139

Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators; Draft Guidance for Industry; Availability AGENCY:

Food and Drug Administration,
HHS.
ACTION:

Notice of availability.

The Food and Drug Administration FDA or Agency is announcing the availability of a draft guidance for industry entitled IND
Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations. This draft guidance provides FDAs recommendations on the chemistry, manufacturing, and controls CMC information needed to support an investigational new drug application IND submitted by a sponsor-investigator developing an individualized antisense oligonucleotide ASO drug product for a severely debilitating or life-threatening SDLT disease caused by a unique genetic variant affecting a small number of individuals typically one or two.
DATES: Submit either electronic or written comments on the draft guidance by February 7, 2022 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
SUMMARY:

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