federal_register: 2021-26453

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2021-26453 Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations; Draft Guidance for Sponsor-Investigators; Availability Notice The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for sponsor-investigators entitled "IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations." FDA is publishing this draft guidance to provide sponsor-investigators (hereafter referred to as sponsors) who are interested in developing individualized antisense oligonucleotide (ASO) drug products for a rapidly progressive, severely debilitating, or life-threatening (SDLT) genetic disease (caused by a unique genetic variant or variants), with clinical recommendations for submission of investigational new drug applications (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. 2021-12-08 2021 12 https://www.federalregister.gov/documents/2021/12/08/2021-26453/investigational-new-drug-application-submissions-for-individualized-antisense-oligonucleotide-drug https://www.govinfo.gov/content/pkg/FR-2021-12-08/pdf/2021-26453.pdf Health and Human Services Department; Food and Drug Administration 221,199 The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for sponsor-investigators entitled "IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or...

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