Argentina has approved Agalzyme, a biosimilar of agalsidase beta, as the first enzyme replacement therapy for Fabry disease in Latin America, representing a major advance in treatment accessibility for this rare disorder.
At an event on 8 August 2025, Laboratorio Biosidus announced the launch of Agalzyme, a biosimilar to Sanofi’s Fabrazyme (agalsidase beta), for the treatment of Fabry disease. This X-linked genetic disorder impairs glycosphingolipid metabolism and has an estimated global birth prevalence of approximately 1 in 15,000, though underdiagnosis suggests the true figure may be higher.
Also known as Anderson–Fabry disease, this rare lysosomal storage disorder results from variants in the GLA gene, which encodes the enzyme alpha-galactosidase A (α-Gal A). Deficient enzyme activity leads to accumulation of globotriaosylceramide (GL-3) in cells—particularly in the kidneys, heart, nervous system, and blood vessels—causing progressive multisystem damage.
Agalsidase beta, the active ingredient in both Fabrazyme and Agalzyme, is an enzyme replacement therapy (ERT) that helps break down accumulated GL-3. It is administered intravenously and is indicated for both adult and paediatric Fabry patients.
Agalzyme is the first biosimilar ERT for Fabry disease approved and launched in Argentina and Latin America. Developed entirely locally using recombinant DNA technology, it offers a safe, effective, and more accessible treatment alternative. An estimated 1,200–1,500 people in Argentina have Fabry disease, though only about 600 currently receive treatment, often due to delayed diagnosis caused by nonspecific symptoms and under recognition.
This approval by Argentina's national regulatory agency (ANMAT) represents a significant milestone for expanding access to high-cost therapies for rare diseases in the Latin American region, which faces challenges such as diagnostic barriers and healthcare system constraints.
Globally, Agalzyme follows the launch of agalsidase beta BS (JR-051), the world’s first biosimilar of agalsidase beta, approved by Japan’s Ministry of Health, Labour and Welfare in September 2018 and subsequently launched in November 2018. JR-051 was developed by JCR Pharmaceuticals in collaboration with Amicus Therapeutics and GlaxoSmithKline.
The biosimilar Agalzyme approval was supported by preclinical data and the Phase III SMILE clinical trial, which met its primary endpoint after 26 weeks and demonstrated therapeutic equivalence to the reference product in terms of efficacy and safety. The trial was conducted at sites in Buenos Aires, Córdoba, and La Rioja. Clinical experts, including Dr Politei, Dr Perretta, Dr Cabrera, and Dr Nieto, have highlighted the therapy’s potential to improve patients’ quality of life and expand treatment access.
In February 2024, mAbxience entered a key manufacturing agreement with Biosidus to support production [1].
The authorization marks a significant advancement in Argentina’s capacity to develop treatments for rare diseases and helps reduce economic barriers to care. Biosidus plans to expand distribution to other emerging markets and eventually pursue regulatory approval in Europe and the US.
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Editor’s comment
European Medicines Agency regulatory requirements ensure the same high standards of quality, safety, and efficacy for biosimilars as for originator biologicals, and also include a rigorous comparability exercise with the reference product but they are not universally accepted by regulatory bodies outside of the European Union (EU). It should be noted that ‘biosimilares’ approved in Argentina might not have been authorized if they had been subjected to the strict regulatory processes required for approval of biosimilars in the EU.
Reference
1. GaBI Online - Generics and Biosimilars Initiative. mAbxience and Biosidus sign an agreement to manufacture Agalsidase Beta[www.gabionline.net]. Mol, Belgium: Pro Pharma Communications International; [cited 2025 Sep 17]. Available from: www.gabionline.net/pharma-news/mabxience-and-biosidus-sign-an-agreement-to-manufacture-agalsidase-beta
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