SIMPATHIC Consortium Advances Drug Repurposing for Rare Diseases
Brussels, Sunday, 13 April 2025.
The SIMPATHIC consortium, backed by Horizon Europe, targets similar pathology mechanisms to repurpose drugs for rare neurological disorders, stepping away from the one-drug-one-disease model.
Innovative Trial Design
The consortium is implementing a groundbreaking basket trial approach that enables testing treatment effectiveness across multiple diseases simultaneously, with sildenafil citrate identified as a promising candidate for clinical testing [1]. This innovative trial design represents a significant departure from traditional methodologies, potentially accelerating the path to market authorization and improving patient access to treatments for rare neurological, neurometabolic, and neuromuscular disorders [1].
Collaborative Framework
The initiative brings together a diverse group of stakeholders, including clinicians, translational researchers, regulatory agencies, and patient representatives, to ensure a robust trial framework [1]. This collaborative approach has led to the development of a master protocol for the basket trial, with disease experts carefully selecting eligible disorders based on comprehensive preclinical evidence [1]. The outcome measure hierarchy has been established through consensus among disease experts, researchers, and patient advocates, ensuring that trial endpoints reflect meaningful clinical benefits [1].
Impact on Rare Disease Treatment
The SIMPATHIC approach is particularly significant given the challenges in developing treatments for rare diseases, where small patient populations often limit trial opportunities [1]. By targeting shared pathomechanisms, the consortium aims to generate high-level evidence on treatment efficacy, even with limited patient numbers [1]. This strategy could prove transformative for patients with rare neurological conditions who traditionally have faced limited treatment options [1].
Future Implications
The consortium’s findings will be presented at the International Drug Repurposing Conference 2025 in Amsterdam on May 7-8, 2025 [1]. This platform will provide an opportunity to share insights from this innovative approach with the broader medical community [1]. With funding from the Horizon Europe research and innovation programme under grant agreement No 101080249, the project represents a significant step forward in addressing the unmet medical needs of rare disease patients [1].