International Exon-Skipping & Gene Therapy Drugs

[Genetic Counselor]

International Exon-Skipping & Gene Therapy Drugs and their availability status in India

[Genetic Counselor]

Several DMD-specific drugs are approved abroad (mainly US FDA) but are not yet approved by CDSCO for the Indian market as of 2026. Listing them here so members don’t confuse “FDA-approved” with “available in India”:

Eteplirsen (Exondys 51) – Exon 51 skipping ASO, FDA accelerated approval (Sarepta).
Golodirsen (Vyondys 53) and Casimersen (Amondys 45) – Exon 53 and Exon 45 skipping ASOs (Sarepta).
Viltolarsen (Viltepso) – Exon 53 skipping ASO.
Delandistrogene moxeparvovec (Elevidys / SRP-9001) – AAV-based micro-dystrophin gene therapy, FDA-approved.
Ataluren (Translarna) – for nonsense-mutation DMD, approved in some markets (not FDA).

Status in India: None currently carry CDSCO marketing approval. Patients/families sometimes explore compassionate-use import licenses via DCGI, but this is costly, case-by-case, and not a substitute for a locally registered trial. Please don’t post anecdotal “it worked for someone I know” claims here without a source — start a separate thread in the support section for personal experiences.

References:

FDA-approved antisense oligonucleotide therapies for DMD: current status and future outlook, https://www.tandfonline.com/doi/full/10.1080/15476286.2026.2689117
FDA Approves SRP-9001 as First Gene Therapy for DMD, https://www.neurologylive.com/view/fda-approves-srp-9001-first-gene-therapy-for-duchenne-muscular-dystrophy

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