FDA Guidance: Human Gene Therapy for Rare Diseases, Retinal Disorder, Neurodegeneration, Bleeding Disorders

 The FDA has issued numerous guidance to facilitate the development of human cell and gene therapy. As the scope of gene therapy has broadened, FDA has outlined additional recommendations pertaining to those disorders. Below are the summary and links for the guidance for developing human gene therapy for a rare disease, neurodegenerative disorders, hemophilia, retinal disorders. 

 
Human Gene Therapy for Rare Diseases
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions
Human Gene Therapy for Neurodegenerative Diseases
Human Gene Therapy for Hemophilia
Human Gene Therapy for Retinal Disorders
Evaluation of Devices Used with Regenerative Medicine Advanced Therapies

Human Gene Therapy for Rare Diseases
This guidance provides recommendations to sponsors developing human gene therapy (GT) products intended to treat a rare disease in adult and/or pediatric patients regarding the manufacturing, preclinical, and clinical trial design issues for all phases of the clinical development program. Such information is intended to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the GT product itself. 
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Expedited Programs for Regenerative Medicine Therapies for Serious Conditions
This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions1 with our recommendations on the expedited development and review of these therapies, including as provided under section 506(g) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as added by section 3033 of the 21st Century Cures Act (Cures Act).2 Under section 506(g) of the FD&C Act, a regenerative medicine therapy can be designated as a regenerative advanced therapy if it meets certain criteria. FDA refers to such designation as “regenerative medicine advanced therapy” (RMAT) designation. (See section III.C of this document). This guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as RMATs. To that end, the guidance provides information about the provisions in the Cures Act regarding the use of the accelerated approval pathway for regenerative medicine therapies that have been granted designation as an RMAT. 

Human Gene Therapy for Neurodegenerative Diseases
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients. Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system. These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. This guidance focuses on considerations for product development, preclinical testing, and clinical trial design. The guidance also discusses marketing approval pathways for investigational gene therapy products.
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Human Gene Therapy for Hemophilia
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for the treatment of hemophilia including clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. This guidance also includes recommendations regarding preclinical considerations to support the development of GT products for the treatment of hemophilia. Additional clinical and preclinical recommendations are available in other guidances (Refs. 1 and 2). This guidance does not provide recommendations for products for the treatment of hemophilia C (factor XI deficiency) or for the treatment of any bleeding disorders other than hemophilia A and B, because of the unique nature of those bleeding disorders.

Human Gene Therapy for Retinal Disorders
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. These disorders vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. These disorders manifest with central or peripheral visual impairment and often with progressive visual loss. This guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products.

Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients. Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system. These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. This guidance focuses on considerations for product development, preclinical testing, and clinical trial design. The guidance also discusses marketing approval pathways for investigational gene therapy products.
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Evaluation of Devices Used with Regenerative Medicine Advanced Therapies
This guidance provides manufacturers, applicants, and sponsors engaged in the development of regenerative medicine therapies, with FDA’s current thinking regarding the evaluation of devices used in the recovery, isolation, or delivery of regenerative medicine advanced therapies

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