Argentinian biotechnology company Biosidus has announced promising interim results from its Phase III SMILE study evaluating a biosimilar version of agalsidase beta for Fabry disease. After 26 weeks of treatment, the study successfully met its primary endpoint, demonstrating the potential of the biosimilar as a safe and effective treatment option.
The complete dataset, including final secondary objectives, is expected to be published in the first half of 2025, with potential commercialization in Argentina beginning by mid-year. The development represents a significant advancement in expanding access to treatment for this rare genetic disorder.
Clinical Trial Results and Development Path
The Phase III SMILE study was specifically designed to assess the comparability between Biosidus's biosimilar and the reference innovator drug. Prior preclinical trials had already confirmed a high degree of similarity with the reference product across all analyzed physicochemical and biological properties.
"This study was designed to scientifically assess the comparability of our drug with the reference innovator drug, analyzing its behavior as a biosimilar," explained Dr. Viridiana Berstein, Clinical Research Manager at Biosidus.
Pharmacokinetic, pharmacodynamic, immunogenic, and safety profiles collected to date suggest comparability to the innovative product. Safety data from the SMILE study continue to support the potential of the drug to become an established biosimilar treatment for Fabry disease.
Market Impact and Expansion Plans
Currently, only two biosimilars of agalsidase beta are available worldwide. Biosidus's product has already received approval in Argentina and awaits final authorization to launch.
"There are currently only two biosimilars of this product available worldwide, and we will have the first one in Argentina and the region," said Mario Koch, Commercial Operations Director for Southern Latam at Biosidus. "Our roadmap for the next two to four years includes strategic entry into emerging markets where Biosidus has established partnerships, with a long-term goal of reaching Europe and the United States. Our vision is to take this product from Argentina to the world."
Addressing Treatment Access Challenges
Fabry disease treatments are typically high-cost therapies that pose significant challenges to healthcare system sustainability and equitable access, particularly in emerging economies. Biosimilars offer a potential solution by providing more affordable alternatives without compromising efficacy or safety.
Koch emphasized the potential impact: "The cost reduction from a biosimilar product, especially for a lifelong treatment, will definitely contribute to the sustainability of the system, probably reducing legal disputes and enabling access to a larger number of patients."
About Biosidus and Its Commitment to Global Access
With over 40 years of experience in biosimilar development, production, and commercialization, Biosidus has established itself as a pioneer in biotechnology research. The company currently exports nine self-developed products to more than 50 countries worldwide.
Biosidus aims to address the significant disparity in global biologics access, where seven countries currently account for 85% of global consumption. Through its biosimilar development program, the company is working to narrow this gap with an ethical and equitable approach to distribution.
About Fabry Disease
Fabry disease is a rare genetic disorder characterized by the buildup of a particular type of fat in the body's cells due to the deficiency of the enzyme alpha-galactosidase A. This accumulation can lead to a range of symptoms affecting multiple organ systems, including the heart, kidneys, and nervous system.
Enzyme replacement therapy with agalsidase beta represents one of the primary treatment approaches for Fabry disease, but its high cost has limited accessibility in many regions. Biosimilar development offers hope for expanded treatment access while maintaining therapeutic efficacy.
