An Illustrative Example from the PaVe-GT Program for Navigating FDA Orphan Drug and Rare Pediatric Disease Designation Applications

被引：0

作者：

Lomash, Richa Madan ^{[1
]}

Shchelochkov, Oleg ^{[2
]}

Chandler, Randy J. ^{[2
]}

Toney, London ^{[1
]}

Portero, Deanna ^{[3
]}

Venditti, Charles P. ^{[2
]}

Pariser, Anne ^{[3
]}

Ottinger, Elizabeth ^{[1
]}

机构：

[1] NCATS, Therapeut Dev Branch, Rockville, MD USA

[2] NIH, Bethesda, MD USA

[3] Natl Ctr Adv Translat Sci NCATS, Bethesda, MD USA

来源：

MOLECULAR THERAPY | 2023年 / 31卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

1658

引用

页码：776 / 777

页数：2

共 6 条

[1] Regulatory Pathway for Gene Therapy for Rare Diseases: An Illustrative Example from the PaVe-GT Platform Program
Stan, Rodica
Lomash, Richa Madan
Chandler, Randy J.
Shchelochkov, Oleg
Venditti, Charles P.
Bonnemann, Carsten G.
Brooks, Philip J.
Ottinger, Elizabeth
MOLECULAR THERAPY, 2023, 31 (04) : 399 - 399
[2] Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
Miller, Kathleen L.
Fermaglich, Lewis J.
Maynard, Janet
ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)
[3] Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
Kathleen L. Miller
Lewis J. Fermaglich
Janet Maynard
Orphanet Journal of Rare Diseases, 16
[4] Correction to: Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development
Catherine Mease
Kathleen L. Miller
Lewis J. Fermaglich
Jeanine Best
Gumei Liu
Erika Torjusen
Orphanet Journal of Rare Diseases, 19
[5] Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience
Lomash, Richa Madan
Shchelochkov, Oleg J.
Chandler, Randy P.
Venditti, Charles A.
Pariser, Anne
Ottinger, Elizabeth
HUMAN GENE THERAPY, 2023, 34 (5-6) : 217 - 227
[6] Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development (vol 19, pg 131, 2024)
Mease, Catherine
Miller, Kathleen L.
Fermaglich, Lewis J.
Best, Jeanine
Liu, Gumei
Torjusen, Erika
ORPHANET JOURNAL OF RARE DISEASES, 2024, 19 (01)

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