SMA therapies outside US/EU - compassionate pathways

What this therapy area looks like

This page is about access to spinal muscular atrophy (SMA). SMA therapies include nusinersen (intrathecal), risdiplam (oral), and onasemnogene abeparvovec (IV gene therapy). Each has distinct access dynamics, and the right-product-for-the-right-patient question is clinical, not commercial.

Why cross-border access comes up

Patients and physicians reach out about spinal muscular atrophy (SMA) when the therapy is not registered in the destination country, when it is registered but unavailable locally, or when the local list price is materially above the US ex-manufacturer price. In each case, the question is whether a Named Patient Program or personal-import pathway in the destination country permits lawful import of a specific quantity for a specific patient, and whether the supply chain from the US source to the treating physician can be coordinated without compromising DSCSA provenance or cold-chain integrity.

Clinical considerations

Access is necessary but not sufficient. For spinal muscular atrophy (SMA), the treating physician is the accountable decision-maker on appropriateness, dosing, and monitoring. A cross-border sourcing partner should never substitute for that judgment. Where relevant, eligibility screening — for example, mutation confirmation, titer testing, weight or age limits, or prior-therapy requirements — must be complete before a shipment is authorized.

Supply and handling

For spinal muscular atrophy (SMA), the practical handling profile typically requires cold-chain (2-8°C) or frozen (−20 to −70°C) storage with documented temperature logs, specialty-pharmacy dispensing under a US-licensed pharmacist, and DSCSA-compliant chain-of-custody from the wholesaler through the final hand-off. Shipments to the destination country are sent under documented carrier protocols to the treating physician or a hospital pharmacy; we do not ship direct-to-patient for these categories.

Pricing and financing

Many spinal muscular atrophy (SMA) therapies carry single-cycle or single-infusion costs that exceed typical out-of-pocket tolerance. Financing options include patient savings, employer health-benefit carve-outs, manufacturer patient-assistance programs, charitable and disease-specific foundations, and in some MENA jurisdictions, sovereign or charitable funds. Reserve Meds can signpost these options; we do not evaluate patient creditworthiness or run financing.

How to start

The fastest path is a short intake at reservemeds.com/request including the drug name, indication, destination country, and a one-line summary of the patient’s clinical status. Our clinical team returns a feasibility note within one business day.

Start a request

Start a request