Protein replacement therapies for rare diseases: a breeze for regulatory approval?
Abstract
Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.
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Many GARDs are characterized by protein deficiencies or malfunctions that require protein replacement therapies.
“Many genetic and rare diseases (GARDs) are characterized by protein deficiencies or malfunctions that require protein replacement therapies (PRTs).[122, 123]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [122]
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“In addition, protein replacement therapies have a higher probability of regulatory approval compared with biologics or small molecules (35).”
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“…1 through the US Food and Drug Administration basic license application approval for a protein replacement therapy reaching the clinic is 88%, and 91% if the therapy is first in class (112), compared with an LOA of 15% for all other molecular entities targeting all other disease entities (113).”
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- Authors
- Gorzelany, J. A., & de Souza, M. P.
- Affiliations
- Laurelwood Biopartners, Boston, MA, 02115, USA.
- Lotus Tissue Repair, a wholly owned subsidiary of Shire Human Genetic Therapies, Lexington, MA 02421, USA.
- Corresponding author
- Gorzelany, Jennifer A.
- Venue
- Science Translational Medicine
- Published
- 2013-03-27
- Volume
- 5
- Issue
- 178
- Pages
- 178fs10-178fs10
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2006476711
- Cited by
- 33
- References
- 9
- Citations by year
- 2026: 2 · 2025: 3 · 2024: 2 · 2023: 8 · 2022: 2 · 2021: 4 · 2020: 2 · 2019: 5 · 2018: 1 · 2017: 2 · 2016: 1 · 2015: 1
- FWCI
- 1.57
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Life Sciences › Immunology and Microbiology › Immunology › Biosimilars and Bioanalytical Methods
- Keywords
- protein replacement therapy, rare diseases, regulatory approval, orphan drugs, monogenic disorders, biologics, small molecules, drug development
- MeSH terms
- Humans, Orphan Drug Production, Proteins, United States, United States Food and Drug Administration, Drug Approval, Rare Diseases, Enzyme Replacement Therapy
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