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# Comments on Neurodegenerative Disease FDA Draft Guidance
- URL: https://www.fdaweb.com/comments-on-neurodegenerative-disease-fda-draft-guidance/
- Published: 2021-04-06T12:00:00.000Z
- Updated: 2026-09-14T16:53:43.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149052

Novartis says an FDA draft guidance, *Human Gene Therapy for Neurodegenerative Diseases*, should leverage past learnings from small molecule, biologic, and current gene therapy experiences to develop a platform approach to support regulatory decision-making. The company comment [letter](https://www.regulations.gov/comment/FDA-2020-D-2101-0008?ref=fdaweb.com) says well-characterized common attributes shared across programs can facilitate the use of a common data set without unnecessary redundant studies. It says a science-based platform approach would enable efficiencies needed to bring more transformative therapies to patients.

In its [comment](https://www.regulations.gov/comment/FDA-2020-D-2101-0005?ref=fdaweb.com), Biogen says that while the draft is a useful overview of high-level recommendations in existing gene therapy guidance, more detailed recommendations are necessary to help guide sponsors as they navigate development. For example, it says, the guidance does not distinguish a rare, rapidly progressing neurological disease from a chronic, slowly progressing disease, although the considerations and rationale may be very different between the two settings. Biogen encourages the agency to develop disease-specific guidance for neurodegenerative diseases that incorporate guidance specific to the development of gene therapy products for those industries. And it supports the use of question-and-answer documents as an alternative approach to inform sponsors of current FDA policy for gene therapies that may not be amenable to the longer lifecycle of guidance development.

Passage Bio makes [recommendations](https://www.regulations.gov/comment/FDA-2020-D-2101-0009?ref=fdaweb.com) on chemistry, manufacturing, and controls, and on considerations for clinical trials.

All three comment letters include detailed, line-by-line technical comments and recommendations.