- In May 2024, Horizon Therapeutics plc, the manufacturer of Procysbi, expanded its global access program to include select regions in Latin America and Asia-Pacific. This strategic move aims to improve treatment availability for underserved cystinosis patients by partnering with local healthcare providers and patient advocacy groups. The initiative underscores Horizon's commitment to equitable access to life-saving therapies for rare disease communities worldwide, further strengthening its position in the global orphan drug market
- In March 2024, Leadiant Biosciences announced the initiation of a post-marketing surveillance study across multiple European countries to assess the long-term safety and efficacy of Cystagon. The study is expected to provide valuable real-world data that will inform future treatment protocols and support regulatory frameworks. This move reflects the growing emphasis on data-driven decision-making in rare disease management and the company’s ongoing investment in improving clinical outcomes for cystinosis patients
- In February 2024, researchers at University College London (UCL), in collaboration with European biotech firms, launched a clinical trial investigating a novel gene therapy targeting the CTNS gene mutation responsible for nephropathic cystinosis. This breakthrough trial represents a significant step toward potentially curative approaches, aiming to restore normal lysosomal function in affected individuals. It highlights the increasing focus on next-generation therapies that go beyond symptom management to address the root cause of the disease
- In January 2024, The Cystinosis Research Foundation awarded new grants totaling over USD 3 million to international research teams focusing on innovative therapies such as mRNA-based treatments, stem cell transplantation, and disease-modifying agents. These funding initiatives aim to accelerate the development of transformative solutions for cystinosis and demonstrate the critical role of nonprofit organizations in driving progress within the rare disease space
- In December 2023, Recordati Rare Diseases announced the expansion of its cystinosis patient support program in Europe and North America, providing resources such as nurse education, home delivery services, and multilingual helplines. This enhancement reflects a growing emphasis on comprehensive care models that improve adherence, quality of life, and treatment outcomes for patients managing chronic rare conditions
