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Exondys 51

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Exondys 51

Supplied with complete CDSCO-approved import and customs documentation, including Form 12A & 12B, CDEC, GST Bill of Entry, Commercial Invoice, and Packing List.

Medicine Name: Exondys 51
Generic Name (API): Eteplirsen
Dosage Form & Strength: 100 mg /2 ml, 500 mg / 10 ml
Route: Intravenous (IV) infusion
Drug Class: Antisense oligonucleotide (exon-skipping therapy)

Manufacturer / Innovator:

USA

Regulatory Approval:

  • US FDA (accelerated approval)
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  • EU-GDP Compliant Warehousing for secure, quality-controlled global distribution.

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  • Temperature-Controlled Logistics for Safe and Timely Delivery

    Temperature-controlled logistics ensuring safe, reliable, and timely medicine delivery.

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What Is Exondys 51 Used For?

Exondys 51 (eteplirsen) is an antisense oligonucleotide that helps skip a mutated section of the DMD gene's pre-mRNA, enabling production of a shorter but functional dystrophin protein. It is indicated for the treatment of:

  • Duchenne muscular dystrophy (DMD), including in pediatric patients, who have a confirmed DMD gene mutation amenable to exon 51 skipping

This indication is approved under accelerated approval based on an increase in dystrophin production observed with treatment; continued approval may depend on results from confirmatory trials. It is a prescription medicine and must be used under the supervision of a qualified neurologist, following genetic confirmation of exon 51-amenable mutation status.

Recommended Dosage

  • 30 mg/kg given as an intravenous infusion once weekly, via an in-line 0.2 micron filter
  • Infused over 35 to 60 minutes

This information is not a treatment plan. For personalized medical advice, consult your doctor and refer to the full prescribing information.

Exondys 51 Warnings and Precautions

  • Kidney toxicity: Based on animal studies, Exondys 51 may affect kidney function. Since it is cleared primarily through the kidneys, patients with mild to moderate kidney impairment may have higher drug exposure, though no specific dose adjustment is currently recommended.
  • Hypersensitivity reactions: Including rash and other allergic reactions, can occur; patients should be monitored during infusions.

How to Access Exondys 51 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 Exondys 51 to India?

Exondys 51 (eteplirsen) 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 51 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 Exondys 51 available in India?

Exondys 51 (eteplirsen injection) is a prescription medicine that legally requires a doctor's prescription to be dispensed. As of current publicly available information, Exondys 51 is not commercially marketed in India.

However, patients in India can legally access Exondys 51 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.

Exondys 51 received US FDA accelerated approval in 2016 for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 51 skipping, ensuring full transparency throughout the process.

Exondys 51 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 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 Exondys 51

Exondys 51 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 Exondys 51 Across Major Cities in India

Named Patient Program supports access to Exondys 51 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 Exondys 51 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 Exondys 51 for eligible DMD patients.

Bangalore

Bangalore's pediatric neurology and genetics centers work with NPP to support patients requiring Exondys 51 for exon 51-amenable DMD.

Chennai

Patients in Chennai can access Exondys 51 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 Exondys 51 through NPP.

Kolkata

NPP facilitates access to Exondys 51 for patients in Kolkata under the guidance of treating pediatric neurologists.

Pune

Patients in Pune can access Exondys 51 through NPP with prescription-based coordination and cold-chain delivery support.

Ahmedabad

NPP supports access to Exondys 51 for patients in Ahmedabad, working directly with treating specialists.

Kochi

Patients in Kochi and across Kerala can access Exondys 51 through NPP's regulated import pathway.

Chandigarh

NPP coordinates access to Exondys 51 for patients in Chandigarh and the surrounding region.

Jaipur

Patients in Jaipur can access Exondys 51 through NPP with support from treating neurologists.

Lucknow

NPP facilitates access to Exondys 51 for DMD patients in Lucknow and across Uttar Pradesh.

Bhubaneswar

Patients in Bhubaneswar can access Exondys 51 through NPP's coordinated import and delivery process.

Guwahati

NPP supports access to Exondys 51 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.

Written By:

Written By: Nitin Goswami, Senior Medical Content Writer
Last Updated: 08 September 2026
Sources: FDA • CDSCO

Medically Reviewed By:

Medically Reviewed By: Shilpi Banerjee, Registered Pharmacist (M.Pharm)
Last Updated: 08 September 2026

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