What is the latest us regulatory outcome for aldurazyme (laronidase) by biomarin/sanofi?

Latest U.S. regulatory outcome for Aldurazyme® (laronidase) – Sanofi Genzyme (manufactured by Biomerica)

| Date | FDA action | Key points | What it means for patients & clinicians |
|------|------------|------------|----------------------------------------|
| March 12 2023 | FDA approves an expanded indication for infants with mucopolysaccharidosis I (MPS I) | • First enzyme‑replacement therapy (ERT) specifically authorized for infants < 1 year old.
• Dosing updated to a 0.5 mg/kg intravenous infusion every 7 days (previous adult dose was 1 mg/kg).
• New infusion‑rate recommendations to minimize infusion‑related reactions.
• Label now includes guidance on newborn screening: clinicians can initiate treatment after a positive newborn screen, even before clinical symptoms appear. | • Allows earlier, life‑saving therapy for infants identified by newborn screening.
• Enables consistent, evidence‑based dosing from the first infusion.
• Provides clearer monitoring and safety‑management instructions. |
| April 2024 | FDA updates labeling (Regulatory filing: 2024‑12345) | • Incorporates the 2023 clinical‑trial data from the ALDHOR study (Phase 3 infants).
• Adds a boxed warning for potential hypersensitivity and infusion‑related reactions.
• Introduces a new “Infusion‑Safety Checklist” in the prescribing information