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Zavesca (miglustat) is an oral substrate reduction therapy used to manage type 1 Gaucher disease in adult patients who cannot receive enzyme replacement therapy.
Zavesca is FDA-approved as monotherapy for adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option, such as due to allergy, hypersensitivity, or poor venous access.
In some countries outside the United States, Zavesca is also approved for Niemann-Pick disease type C. This is not an FDA-approved indication, and this distinction is disclosed here for full transparency.
Miglustat inhibits glucosylceramide synthase, an enzyme involved in the production of glycosphingolipids, reducing the substrate buildup that characterizes Gaucher disease.
The recommended dose for adults with type 1 Gaucher disease is one 100 mg capsule taken orally three times a day. Patients with moderate renal impairment start at a reduced dose of one 100 mg capsule per day, as determined by the treating specialist.
Zavesca requires monitoring for several important safety considerations.
The most common serious adverse reaction. Patients should promptly report any numbness, tingling, pain, or burning in the hands and feet, and nerve conduction studies are recommended at baseline and periodically thereafter.
Gastrointestinal effects, including diarrhea and weight loss, are common and can be serious in some patients.
Can occur, particularly early in treatment; usually resolves with dose reduction.
Monitor platelet counts periodically during treatment.
Zavesca can cause fetal harm. Effective contraception is required during treatment and for a defined period after the final dose.
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 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.
Zavesca (miglustat) received FDA approval in 2003 and is manufactured by Janssen, for adult patients with type 1 Gaucher disease who cannot receive enzyme replacement therapy.
Import of Zavesca through a Named Patient Program requires a valid prescription from the treating metabolic specialist, confirmation of Gaucher disease diagnosis, and supporting clinical documentation as required by Indian regulatory authorities.
NPP sources Zavesca directly from Janssen's authorized supply chain to ensure product authenticity and regulatory compliance.
Zavesca is sourced through verified, GDP-compliant channels from Janssen's authorized supply chain to ensure authenticity and quality.
Zavesca capsules should be stored at 20°C to 25°C (68°F to 77°F), with excursions permitted between 15°C and 30°C (59°F to 86°F).
Once sourced, Zavesca is packed and transported under appropriate temperature-controlled 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 Zavesca through NPP's regulated import pathway, with metabolic disease specialists in major cities supporting the treatment process.
Leading metabolic disease centers in Mumbai support Gaucher disease diagnosis and treatment coordination for Zavesca access.
Delhi's major hospitals assist patients with diagnostic confirmation and prescription support for Zavesca.
Bangalore's specialist network offers access to Gaucher disease treatment planning for eligible patients.
Chennai-based specialists coordinate Zavesca access alongside comprehensive metabolic disease care.
Hyderabad's care institutions support patients pursuing Zavesca therapy.
Kolkata's specialist centers help coordinate documentation and treatment access for Zavesca.
Pune-based specialists assist with diagnosis confirmation and Zavesca treatment planning.
Ahmedabad's specialty hospitals support the import and treatment process for Zavesca.
Kochi's specialist units assist patients with the documentation needed for Zavesca access.
Chandigarh's care centers support Zavesca treatment coordination for patients from North India.
Jaipur's specialists help patients navigate the Zavesca access process.
Lucknow-based centers assist patients pursuing Zavesca therapy.
Bhubaneswar's institutions support access to Zavesca for eligible patients.
Guwahati's specialists help coordinate Zavesca access for patients across the Northeast.
Patients in other cities across India can also access Zavesca 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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