Vyondys 53

Vyondys 53 Warnings and Precautions

• Kidney toxicity: Based on animal data, Vyondys 53 may cause kidney toxicity. Although kidney toxicity was not observed in clinical studies with Vyondys 53, potentially fatal glomerulonephritis has been observed with some other antisense oligonucleotides, so kidney function should be monitored during treatment.
• Kidney function monitoring: Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting treatment, with glomerular filtration rate assessment considered. Creatinine may not reliably measure kidney function in DMD patients.
• Hypersensitivity reactions: Including rash and other allergic reactions, can occur; patients should be monitored during infusions.

How to Access Vyondys 53 in India: A 6-Step Process

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.

Documentation & Availability

What documents are required to import Vyondys 53 to India?

Vyondys 53 (golodirsen) injection may be imported in the patient's name only, by patients or government hospitals, in line with India's import regulations for unapproved medicines. The following documentation is required:

• A valid prescription from a qualified neurologist
• The patient's diagnostic reports, including genetic confirmation of a DMD gene mutation amenable to exon 53 skipping
• Patient ID proof (issued by the Government of India)

How is the order confirmed?

The order is confirmed only after receipt and verification of a valid prescription from the doctor and, if applicable, an import permit.

Is Vyondys 53 available in India?

Vyondys 53 (golodirsen injection) is a prescription medicine that legally requires a doctor's prescription to be dispensed. As of current publicly available information, Vyondys 53 is not commercially marketed in India.

However, patients in India can legally access Vyondys 53 through the Named Patient Program (NPP) — an import provision that allows patients to receive medicines approved in other countries but not yet marketed in India, under a doctor's prescription and proper regulatory documentation.

Vyondys 53 received US FDA accelerated approval in December 2019 for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping, ensuring full transparency throughout the process.

Vyondys 53 can be made available to patients, doctors, and hospitals across major cities in India, including Gurgaon, Delhi, Bangalore, Mumbai, Navi Mumbai, Thane, Hyderabad, Mysore, Kerala, Chennai, Coimbatore, Madurai, Kolkata, Ahmedabad, Surat, Vadodara, Pune, Nagpur, Nashik, Jaipur, Udaipur, Lucknow, Kanpur, Gorakhpur, Chandigarh, Mohali, Faridabad, Noida, Ghaziabad, Kochi, Trivandrum, Kozhikode, Thrissur, Kannur, Mangalore, Bhubaneswar, Patna, Ranchi, Raipur, Guwahati, Visakhapatnam, Vijayawada, Goa, Jammu, Srinagar, Dehradun, Bhopal, Indore, Ludhiana, Amritsar, and others.

NPP also facilitates access to other exon-skipping therapies for Duchenne muscular dystrophy, including Exondys 51 and Amondys 45, for patients whose treating neurologist considers these options relevant to their case based on the patient's specific DMD gene mutation.

Sourcing & Delivery of Vyondys 53

Vyondys 53 is sourced directly from authorized channels in the country of origin and shipped to India under GDP-compliant, temperature-controlled logistics to preserve product integrity from dispatch to delivery.

• Cold-chain shipping maintained at 2°C to 8°C throughout transit
• Real-time shipment tracking and delivery updates
• Delivery coordinated directly with the patient, family, or treating hospital

Availability of Vyondys 53 Across Major Cities in India

Named Patient Program supports access to Vyondys 53 for patients with Duchenne muscular dystrophy across major cities in India, coordinating documentation, sourcing, and delivery with treating neurologists.

Mumbai

Patients in Mumbai can access Vyondys 53 through NPP with support from treating neurologists at the city's leading neuromuscular and pediatric neurology centers.

Delhi

In Delhi, NPP coordinates with hospitals and specialty neurology clinics to facilitate timely access to Vyondys 53 for eligible DMD patients.

Bangalore

Bangalore's pediatric neurology and genetics centers work with NPP to support patients requiring Vyondys 53 for exon 53-amenable DMD.

Chennai

Patients in Chennai can access Vyondys 53 through NPP, with coordination between treating doctors and the program's regulatory and logistics team.

Hyderabad

Hyderabad's neurology and genetic testing centers support diagnosis and access coordination for Vyondys 53 through NPP.

Kolkata

NPP facilitates access to Vyondys 53 for patients in Kolkata under the guidance of treating pediatric neurologists.

Pune

Patients in Pune can access Vyondys 53 through NPP with prescription-based coordination and cold-chain delivery support.

Ahmedabad

NPP supports access to Vyondys 53 for patients in Ahmedabad, working directly with treating specialists.

Kochi

Patients in Kochi and across Kerala can access Vyondys 53 through NPP's regulated import pathway.

Chandigarh

NPP coordinates access to Vyondys 53 for patients in Chandigarh and the surrounding region.

Jaipur

Patients in Jaipur can access Vyondys 53 through NPP with support from treating neurologists.

Lucknow

NPP facilitates access to Vyondys 53 for DMD patients in Lucknow and across Uttar Pradesh.

Bhubaneswar

Patients in Bhubaneswar can access Vyondys 53 through NPP's coordinated import and delivery process.

Guwahati

NPP supports access to Vyondys 53 for patients in Guwahati and across the Northeast region.

NPP also supports access for patients in other Indian cities not listed above — contact the team to check availability and timelines for your location.