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Vimizim (elosulfase alfa) is the first and only enzyme replacement therapy approved for mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, a rare genetic disorder caused by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase.
Vimizim is FDA-approved for patients with MPS IVA to replace the deficient enzyme and reduce the buildup of glycosaminoglycans that causes progressive skeletal, respiratory, and other organ system damage.
Elosulfase alfa is a recombinant form of the enzyme N-acetylgalactosamine-6-sulfatase, which patients with MPS IVA lack or have in insufficient amounts. Replacing this enzyme helps break down glycosaminoglycans that would otherwise accumulate in tissues throughout the body.
The recommended dose is 2 mg/kg administered by intravenous infusion once weekly, typically over 3.5 to 4.5 hours, as determined by the treating metabolic specialist.
Boxed Warning — Vimizim: Patients treated with Vimizim have experienced life-threatening hypersensitivity reactions, including anaphylaxis, which can occur during early treatment or after extended duration of therapy — cases have been reported as early as 30 minutes into the first infusion and as late as the 47th infusion. Vimizim should be administered in a healthcare setting with appropriate medical support and monitoring, including access to cardiopulmonary resuscitation equipment and epinephrine.
Monitor closely during and after each infusion. Discontinue immediately and initiate appropriate treatment if severe hypersensitivity or anaphylaxis occurs.
Patients with compromised respiratory or cardiac function may be at increased risk during infusion; monitor closely.
MPS IVA patients are at risk for spinal or cervical cord compression, which requires ongoing monitoring by the treating specialist independent of Vimizim treatment.
Step 1 – Search & request your medicine: The patient or treating doctor submits a request with basic details to initiate the access process.
Step 2 – Verification & eligibility review: The team verifies availability and reviews eligibility against regulatory and clinical requirements to ensure a compliant pathway.
Step 3 – Receive quote & delivery timeline: A transparent quotation is shared, covering medicine cost, logistics, and a clear delivery timeline.
Step 4 – Regulatory approval process: Assistance is provided to secure the approvals and documentation required for legal cross-border access.
Step 5 – Temperature-controlled import & handling: The medicine is sourced and transported under strict GDP-compliant, cold-chain conditions to maintain product integrity.
Step 6 – Secure delivery: The medicine is delivered to the patient or treating hospital, with updates provided at each stage.
Note: From request to delivery, every step is designed to ensure your treatment reaches you safely, compliantly, and without unnecessary delays.
Vimizim (elosulfase alfa) is manufactured by BioMarin, the first and only enzyme replacement therapy approved for MPS IVA (Morquio A syndrome).
Import of Vimizim through a Named Patient Program requires a valid prescription from the treating metabolic specialist, confirmation of MPS IVA diagnosis, and supporting clinical documentation as required by Indian regulatory authorities.
NPP sources Vimizim directly from BioMarin's authorized supply chain to ensure product authenticity and regulatory compliance.
Vimizim is sourced through verified, GDP-compliant, cold-chain channels from BioMarin's authorized supply chain to ensure authenticity and quality.
Vimizim should be stored refrigerated at 2°C to 8°C, in the original carton to protect from light. Do not freeze.
Once sourced, Vimizim is packed and transported under continuous cold-chain conditions to preserve product integrity throughout the import process, with tracking and updates provided at each stage of delivery to the patient or treating hospital in India.
Patients across India can access Vimizim through NPP's regulated import pathway, with metabolic disease specialists in major cities supporting the treatment process.
Leading metabolic disease centers in Mumbai support MPS IVA diagnosis and treatment coordination for Vimizim access.
Delhi's major hospitals assist patients with diagnostic confirmation and prescription support for Vimizim.
Bangalore's specialist network offers access to MPS IVA treatment planning for eligible patients.
Chennai-based specialists coordinate Vimizim access alongside comprehensive metabolic disease care.
Hyderabad's care institutions support patients pursuing Vimizim therapy.
Kolkata's specialist centers help coordinate documentation and treatment access for Vimizim.
Pune-based specialists assist with diagnosis confirmation and Vimizim treatment planning.
Ahmedabad's specialty hospitals support the import and treatment process for Vimizim.
Kochi's specialist units assist patients with the documentation needed for Vimizim access.
Chandigarh's care centers support Vimizim treatment coordination for patients from North India.
Jaipur's specialists help patients navigate the Vimizim access process.
Lucknow-based centers assist patients pursuing Vimizim therapy.
Bhubaneswar's institutions support access to Vimizim for eligible patients.
Guwahati's specialists help coordinate Vimizim access for patients across the Northeast.
Patients in other cities across India can also access Vimizim through NPP's nationwide coordination network.
Written By: Nitin Goswami, Senior Medical Content Writer
Last Updated: 10 September 2026
Sources: FDA • CDSCO
Medically Reviewed By: Shilpi Banerjee, Registered Pharmacist (M.Pharm)
Last Updated: 10 September 2026
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