Sarepta: cross-border access for the specialty portfolio

Quick orientation

Sarepta Therapeutics, Inc. is a US-headquartered biotechnology company based in Cambridge, Massachusetts. Sarepta is the leading commercial-stage company in Duchenne muscular dystrophy (DMD) therapeutics. The specialty footprint comprises three exon-skipping antisense oligonucleotide products targeting specific dystrophin mutations (Exondys 51 for exon-51-amenable mutations, Vyondys 53 for exon-53-amenable, Amondys 45 for exon-45-amenable), and the AAV-based gene therapy Elevidys for ambulatory and non-ambulatory DMD patients age 4 and older.

For Reserve Meds cross-border named-patient programs, the Sarepta DMD franchise is one of the highest-acuity catalogs: every product targets a population where the alternative is supportive care plus corticosteroids only, and local registration in MENA and India is the exception rather than the rule.

Portfolio in cross-border NPP scope

Elevidys (delandistrogene moxeparvovec-rokl)

AAV9-based gene therapy approved for Duchenne muscular dystrophy in ambulatory and non-ambulatory patients age 4 and older with a confirmed DMD gene mutation. Single-infusion gene therapy. US WAC at single-dose administration runs into the multi-million-dollar range. Reserve Meds coordinates Elevidys on a case-by-case basis because the single-infusion gene therapy is administered at a US-authorized treatment center. The Reserve Meds coordinator works with the patient on pre-trip preparation including AAVrh74 antibody screening and the US treatment center introduction.

Exondys 51 (eteplirsen)

Phosphorodiamidate morpholino antisense oligonucleotide approved for DMD in patients with a confirmed mutation amenable to exon 51 skipping. Administered as weekly intravenous infusion. International requests come from DMD patients in MENA and India with confirmed exon-51-amenable mutations where the family has chosen the exon-skipping pathway over the Elevidys gene-therapy pathway, or where the patient is outside the Elevidys eligibility criteria.

Vyondys 53 (golodirsen)

Phosphorodiamidate morpholino antisense oligonucleotide approved for DMD in patients with a confirmed mutation amenable to exon 53 skipping. Administered as weekly intravenous infusion. International requests follow the same pattern as Exondys 51 for the exon-53-amenable subgroup.

Amondys 45 (casimersen)

Phosphorodiamidate morpholino antisense oligonucleotide approved for DMD in patients with a confirmed mutation amenable to exon 45 skipping. Administered as weekly intravenous infusion. International requests come from the exon-45-amenable DMD subgroup.

Viltepso (viltolarsen, co-marketed with NS Pharma)

Phosphorodiamidate morpholino antisense oligonucleotide approved for DMD in patients with a confirmed mutation amenable to exon 53 skipping. Sarepta does not directly market Viltepso (which is the NS Pharma product), but Reserve Meds intake notes that DMD families often compare Viltepso and Vyondys 53 head-to-head for the exon-53 patient population.

Why patients route Sarepta products through cross-border NPP

The Sarepta cross-border driver is structural. First, Duchenne muscular dystrophy mutation-specific exon-skipping therapies serve sub-populations of an already rare disease. Each of Exondys 51, Vyondys 53, and Amondys 45 is approved only for a specific subset of the DMD population, and the locally registered options in MENA and India are typically corticosteroids only.

Second, Elevidys gene therapy is the largest disease-modifying option for ambulatory DMD patients age 4 and older, but the medical-travel logistics and the AAVrh74 antibody screening requirement add complexity that benefits from coordinated cross-border support.

Third, the Sarepta SareptAssist patient support program is restricted to US residents. International patients route as cash-pay through the named-patient pathway.

Manufacturer engagement posture

Reserve Meds coordinates Sarepta-manufactured US product through DSCSA-compliant specialty wholesaler channels. We are not an authorized Sarepta distributor. All Reserve Meds orders are cash-pay at firm-quote pricing.

For Sarepta market access teams: Reserve Meds maintains structured demand visibility across the exon-skipping antisense oligonucleotide population and the Elevidys medical-travel pipeline. If your group is sizing pre-registration demand in MENA and India for the DMD franchise specifically, or if your group is sizing the Elevidys medical-travel international pipeline, we are open to a structured conversation under NDA.

Common cross-border destinations for the Sarepta portfolio

The DMD exon-skipping franchise concentrates in pediatric neuromuscular centers in the UAE, Saudi Arabia, Egypt, and India where DMD patient registries are established and where consanguinity-driven prevalence is meaningful. Elevidys medical-travel requests originate from the same neuromuscular centers, with families typically planning the US treatment-center visit 3-6 months in advance to complete the AAVrh74 antibody screening and pre-treatment workup.

What Reserve Meds provides

For every Sarepta product order, Reserve Meds coordinates DSCSA chain-of-custody documentation; cold-chain validated logistics where required; country regulatory pathway expertise covering named-patient import approval, customs clearance, and any required physician declaration; and a single patient-facing coordinator. We do not replace the patient's treating physician. A US-licensed pharmacist reviews every prescription before dispensing.

Next step for patients

Patients or caregivers who have a treating physician's prescription for a Sarepta product and want a firm quote can start a request below.

Start a request for a Sarepta product

Next step for Sarepta teams

If you are on the Sarepta access, medical affairs, or international market access team and want to discuss coordinated named-patient program demand from MENA and India, reach us at [email protected]. We will share aggregated demand views under NDA.

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