BIO Comments on FDA’s Draft Guidance on Human Gene Therapy for Neurodegenerative Diseases
April 6, 2021
On Tuesday, April 6th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s January 2021 draft industry guidance on “Human Gene Therapy for Neurodegenerative Diseases.” In its comments, BIO stated that while the guidance covers many critical development issues for gene therapies, it is not specific to gene therapies for neurodegenerative diseases, and further requests that the Agency consider the potential impact of this guidance on gene therapy programs outside of neurodegenerative diseases. To this end, BIO encourages the issuance of (1) broad guidance on gene therapies that includes information that is applicable across diseases and (2) publication of focused and brief disease-specific guidance to provide information to Sponsors on disease-specific issues, as appropriate. Since aspects of this guidance may be relevant to cell therapies, BIO recommends that FDA develop a guidance or Q&A document that is quickly and easily updated, and consolidates cell and gene therapy policy with broad applicability into a single document. In the full comment letter, BIO also provides specific comments on the following topics: Gene Therapies and Accelerated Approval, Preclinical Studies, Chemistry Manufacturing and Controls (CMC), and Clinical Studies.
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BIO Comments on FDA’s Draft Guidance on Human Gene Therapy for Neurodegenerative Diseases
The Biotechnology Innovation Organization appreciates the opportunity to comment on the Center for Medicare and Medicaid Services’ Information Collection Request on the Part C and Part D Medicare Prescription Payment Plan Model Documents.
The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the request and comments on the Advanced Manufacturing Technologies Designation Program Draft Guidance.
On Tuesday, April 6th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s January 2021 draft industry guidance on “Human Gene Therapy for Neurodegenerative Diseases.” In its comments, BIO stated that while the guidance covers many critical development issues for gene therapies, it is not specific to gene therapies for neurodegenerative diseases, and further requests that the Agency consider the potential impact of this guidance on gene therapy programs outside of neurodegenerative diseases. To this end, BIO encourages the issuance of (1) broad guidance on gene therapies that includes information that is applicable across diseases and (2) publication of focused and brief disease-specific guidance to provide information to Sponsors on disease-specific issues, as appropriate. Since aspects of this guidance may be relevant to cell therapies, BIO recommends that FDA develop a guidance or Q&A document that is quickly and easily updated, and consolidates cell and gene therapy policy with broad applicability into a single document. In the full comment letter, BIO also provides specific comments on the following topics: Gene Therapies and Accelerated Approval, Preclinical Studies, Chemistry Manufacturing and Controls (CMC), and Clinical Studies.