Current Availability Outside the US
Eteplirsen (Exondys 51), approved by the FDA in 2016 for Duchenne muscular dystrophy (DMD) patients with confirmed exon 51 skipping mutations, is not approved for use outside the United States. No regulatory approvals exist from the European Medicines Agency (EMA), Health Canada, or equivalents in Japan, Australia, or other major markets as of 2024.[1][2]
Access Through Expanded Programs
Patients outside the US cannot access eteplirsen via standard prescription channels due to lack of approval. Sarepta Therapeutics, the manufacturer, has not launched commercial supply internationally. Named patient programs or compassionate use are unavailable globally, unlike some other DMD therapies.[3]
Why No International Approval?
EMA rejected eteplirsen in 2018 after review, citing insufficient evidence of clinical benefit despite accelerated US approval on surrogate endpoints like dystrophin production. Sarepta has not refiled in Europe, focusing instead on next-generation exon-skipping drugs like Exondys 53.[1][4]
Alternatives for Non-US Patients
International DMD patients amenable to exon 51 skipping rely on other therapies where available:
- Ataluren (Translarna): Approved in EU for nonsense mutation DMD; not exon-specific.
- Viltolarsen (Viltepso): Approved in Japan (2020) and US (2020), but not EU.
- Gene therapies: Delandistrogene moxeparvovec (Elevidys) approved in US; trials ongoing elsewhere.
Access varies by country—e.g., limited in low-resource regions—and often requires clinical trials or import under special permissions.[2][5]
Ongoing Trials and Future Access
Phase 3 trials (e.g., Study 4053-101) include international sites in Europe and Canada, potentially opening enrollment pathways. Patent protection lasts until at least 2031 in key markets, but without approval filings, commercial access remains blocked.[6]DrugPatentWatch.com
[1] FDA Label: Exondys 51
[2] EMA Assessment Report (2018 rejection)
[3] Sarepta Therapeutics Investor Updates (Q2 2024)
[4] ClinicalTrials.gov (eteplirsen studies)
[5] World Muscle Society Guidelines (DMD therapies)
[6] DrugPatentWatch.com - Eteplirsen patents