Investigating the Potential for Repurposing Drugs to Treat Rare Diseases

OBJECTIVES: This study aims to explore the potential of drug repurposing as a strategic approach to address unmet medical needs in rare diseases, specifically focusing on Spinal Muscular Atrophy (SMA). The primary objective is to share insights derived from a comprehensive database of FDA-approved Biologics License Applications (BLAs) and New Drug Applications (NDAs) developed by Sanae Akodad, detailing repurposing targets, approval dates, and pharmaceutical companies involved.

METHODS: A comprehensive database was created, encompassing FDA-approved BLAs and NDAs from 1985 to 2024. The database includes detailed information on repurposing targets, approval dates, and the pharmaceutical companies involved. This study utilized this database to conduct an extensive analysis of drug repurposing trends, identifying key industry players and examining the benefits and challenges associated with repurposing drugs for rare diseases. Specific focus was placed on SMA as a case study to highlight the practical application and impact of drug repurposing.

RESULTS: The analysis revealed significant trends and strategic shifts within the pharmaceutical industry towards drug repurposing. Notably, the average time interval between initial FDA approval and subsequent repurposing approval was approximately 8 years. The study identified major pharmaceutical companies leading repurposing initiatives and highlighted the increasing reliance on repurposing strategies over de novo drug development. Additionally, the database showed that orphan drugs initially targeting rare diseases had a significant repurposing rate for other indications, with 27.08% of such drugs being repurposed.

CONCLUSIONS: The comprehensive database developed serves as a critical tool for understanding and advancing drug repurposing strategies. The findings underscore the potential of drug repurposing to develop effective treatments more rapidly and affordably, enhancing accessibility to medical solutions for rare diseases like SMA. This research emphasizes the importance of methodologically robust databases in facilitating strategic shifts within the pharmaceutical industry and addressing critical health needs.