India's Central Drugs Standard Control Organisation (CDSCO) Subject Expert Committee (SEC) has approved prescribing information updates for two of Sanofi's rare disease enzyme replacement therapies, marking important regulatory milestones for patients with lysosomal storage disorders in the country.
Fabrazyme Label Update Approved with Additional Requirements
The SEC approved an updated prescribing information for Fabrazyme (agalsidase beta), used to treat Fabry disease, during its Endocrinology and Metabolism meeting on April 22, 2025. The updated prescribing information, dated October 2024, aligns with Sanofi's Company Core Data Sheet (CCDS) versions 5 (May 6, 2021), 6 (November 18, 2021), and 7 (July 20, 2023).
Fabrazyme contains agalsidase beta, a recombinant form of the human enzyme alpha-galactosidase A. The therapy addresses Fabry disease, a rare genetic disorder characterized by the accumulation of globotriaosylceramide (GL-3) in various body tissues. Administered via intravenous infusion, the enzyme replacement therapy helps break down GL-3 deposits and reduces the disease's impact on critical organs including the kidneys and heart.
The committee has directed Sanofi to submit the European Medicines Agency (EMA) approval of the revised prescribing information to CDSCO for additional evaluation, indicating a requirement for further regulatory harmonization.
Aldurazyme Receives Approval for US-Aligned Labeling
In a separate decision during the same meeting, the SEC recommended approval for updated prescribing information for Aldurazyme (laronidase). This approval follows Sanofi's submission seeking to align the Indian prescribing information with the US Prescribing Information (USPI) dated December 2023.
Aldurazyme provides enzyme replacement therapy for patients with Mucopolysaccharidosis I (MPS I), a rare inherited lysosomal storage disorder. The therapy contains laronidase as its active ingredient and is administered intravenously to manage non-neurological symptoms associated with MPS I.
The treatment addresses clinical manifestations including enlarged liver, joint stiffness, and reduced lung function, which result from the pathological accumulation of glycosaminoglycans (GAGs) in body tissues. Laronidase functions through cellular uptake via mannose-6-phosphate (M6P) receptors, enabling the enzyme to reach lysosomes where it breaks down accumulated GAGs, thereby reducing disease burden.
Regulatory Significance for Rare Disease Management
These approvals represent important steps in maintaining current prescribing standards for rare disease therapies in India. Both Fabry disease and MPS I are ultra-rare genetic disorders with significant unmet medical needs, making access to updated therapeutic information crucial for healthcare providers managing these complex conditions.
The harmonization of prescribing information across different regulatory jurisdictions helps ensure that Indian healthcare professionals have access to the most current safety and efficacy data, supporting optimal patient care in the rare disease space. The requirement for additional EMA documentation for Fabrazyme suggests CDSCO's commitment to comprehensive regulatory review processes for these specialized therapies.
