Repurposing Drugs for Orphan Diseases
C Fratazzi* and J Niu
BBCR Consulting, One Broadway, Cambridge, MA, USA
*Corresponding Author: C Fratazzi, BBCR Consulting, One Broadway, Cambridge, MA, USA.
December 08, 2021; Published: December 31, 2021
Drug repurposing, also known as drug repositioning or reprofiling, develops and commercializes new uses for existing or abandoned drugs. In past decades, ~30% of repurposed drugs were approved by the United States Food and Drug Administration (FDA), while less than 10% of de novo drug applications achieved approval.
Repurposing drugs were developed for various diseases, including cancer, infection, inflammatory disorders, and neurodegenerative diseases. Experimental and computational strategies have been applied in drug repurposing. Professional expert guidance is an essential support for developing a successful regulatory and clinical drug repurposing plan. Our knowledge and experience in regulatory, clinical research, and biomarker consulting provide high-value and support our clients' operational and functional needs. The opportunity to support clients' implementing the most appropriate FDA 505(b)(2) and EMA hybrid submissions maximizes the benefit of drug repurposing for orphan indications. It helps patients with rare diseases and the healthcare system.
This review focuses on developing repurposing drugs for orphan indications and rare diseases. Due to the significant complexity of orphan drug development and lack of effective treatment for many of them, we aim to provide options to address the current unmet medical needs for a wide range of patients worldwide.
Keywords: Drug repurposing (DR); Food and Drug Administration (FDA); Drugs
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