PTC Therapeutics: cross-border access for the specialty portfolio

Quick orientation

PTC Therapeutics, Inc. is a US biopharmaceutical company headquartered in Warren, New Jersey. PTC is focused on rare disease and oncology, with a particular strategic concentration in pediatric neurology and metabolic rare disease. The company trades on NASDAQ as PTCT. FDA approval scale spans multiple marketed products including the SMA franchise asset Evrysdi (partnered with Roche), Emflaza for Duchenne muscular dystrophy, and Upstaza (eladocagene exuparvovec) gene therapy for AADC deficiency.

PTC portfolio in cross-border NPP scope

Evrysdi (risdiplam), partnered with Roche/Genentech, is an FDA-approved oral SMN2 splicing modifier for spinal muscular atrophy in adults and pediatric patients two months of age and older (FDA approval August 2020). Evrysdi is the first oral SMA therapy and addresses a major unmet need for SMA Type 1, 2, and 3 patients across the age spectrum. Daily oral dosing as a powder for reconstitution into a liquid. US list pricing is weight-based and ranges from approximately US$100,000 to US$340,000 per year. The product requires refrigerated storage and is shipped cold-chain.

Emflaza (deflazacort) is an FDA-approved corticosteroid for Duchenne muscular dystrophy (DMD) in patients five years of age and older. Daily oral dosing. US list pricing varies but is approximately US$90,000-$110,000 per year at standard dosing. Emflaza is FDA-approved with a labeled DMD indication, distinguishing it from prednisone/prednisolone which is used off-label for DMD.

Translarna (ataluren) is an oral nonsense-mutation suppression therapy for nonsense mutation DMD. Translarna is not FDA-approved in the United States; FDA application was withdrawn following a 2017 Complete Response Letter and subsequent regulatory engagement. Translarna is approved in the European Union under conditional marketing authorisation. Reserve Meds does not source Translarna from US channels because it is not FDA-approved.

Upstaza (eladocagene exuparvovec) is an AAV gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. Upstaza is approved by the European Commission and is delivered by intraputaminal injection; it is not currently FDA-approved in the United States. Reserve Meds does not source Upstaza from US channels.

Tegsedi (inotersen) was previously commercialised for hereditary transthyretin amyloidosis through PTC's commercial arrangements; we direct hATTR patients to current standard-of-care therapies through their treating physician.

Why patients route through cross-border NPP

Evrysdi is the highest-volume cross-border NPP demand driver in the PTC portfolio. Spinal muscular atrophy is a rare autosomal recessive neuromuscular disease, and the SMA therapy landscape includes three approved options globally: Spinraza (intrathecal antisense), Zolgensma (one-time AAV gene therapy), and Evrysdi (oral). Evrysdi's oral administration is a major access advantage for patients in jurisdictions where intrathecal or specialised AAV infusion capability is limited. Outside the United States, Evrysdi is approved in many markets through the Roche commercialisation partnership, but country-by-country reimbursement and stocking remain uneven across the GCC, India, parts of MENA, and Southeast Asia.

Emflaza for DMD addresses a separate cross-border demand. While prednisone and prednisolone are widely available and used off-label for DMD, Emflaza's pharmacokinetic and side-effect profile is preferred by many neuromuscular specialists and is the only FDA-approved corticosteroid labeled for DMD. Families seeking the FDA-labeled product where local supply is unavailable route through Reserve Meds.

Common cross-border destinations

The highest-frequency NPP destinations for PTC products from US-approved inventory are India, the United Arab Emirates, Saudi Arabia, Qatar, and Turkey. Pediatric neurology and neuromuscular centers in Mumbai, Bangalore, Delhi, Hyderabad, Dubai, Abu Dhabi, Riyadh, and Istanbul drive most inbound referrals.

Manufacturer engagement posture

Reserve Meds coordinates PTC-sourced US commercial product through DSCSA-compliant US specialty distributors for FDA-approved products only. We do not source Translarna or Upstaza from US channels because they are not FDA-approved. We are not affiliated with PTC Therapeutics or its commercial partners. For PTC medical affairs or US commercial teams interested in cross-border NPP pathways, our BD contact is [email protected].

What Reserve Meds provides

For each PTC product request, Reserve Meds coordinates: prescription verification with the treating pediatric neurologist or neuromuscular specialist; genetic confirmation review (SMN1 deletion for Evrysdi; DMD gene mutation analysis for Emflaza); DSCSA serialized chain-of-custody from PTC or partner through licensed US wholesalers to the named patient; refrigerated cold-chain shipping (2 to 8 degrees C) for Evrysdi with temperature data-loggers; ambient shipping for Emflaza tablets; destination-country regulatory documentation for Ministry of Health named-patient approvals and customs clearance; and a dedicated coordinator from prescription to delivery.

Clinical governance and prescription review

Every prescription for a product on this page is reviewed by a US-licensed pharmacist before dispensing. Reserve Meds does not substitute generic equivalents for branded specialty products without an explicit prescriber instruction, and we do not dispense any product whose chain-of-custody we cannot serialize back to the FDA-authorised manufacturer or its licensed wholesale partner. Our clinical review confirms indication-on-label appropriateness based on the documentation submitted by the treating physician, identifies any standard drug-drug interaction or contraindication concerns, and confirms that the requested dose, dosing schedule, and dosage form match the prescription. Where the prescription is unclear or where the indication on the prescription is off-label, our pharmacist contacts the prescriber directly. We do not provide medical advice to patients beyond drug-information counseling at dispensing.

Supply chain integrity

Supply integrity is the foundation of Reserve Meds operations. Every shipment carries a serialized DSCSA pedigree document confirming the chain of ownership from the FDA-registered manufacturer or its authorised distributor through a licensed US wholesale distributor to Reserve Meds and onward to the named patient. We do not source through parallel-import channels, third-country re-import, or unverified online wholesale markets. Cold-chain shipments are packed in qualified shippers with temperature data-loggers reading and logging temperature throughout the journey; data-logger reads are reviewed on arrival, and any out-of-specification excursion triggers our quality-hold protocol. For frozen products such as gene therapies and certain antibody-drug conjugates, we use validated frozen-shipper systems with dry-ice replenishment as required.

Destination-country regulatory pathways

Cross-border named patient access operates under jurisdiction-specific regulatory frameworks. In the United Arab Emirates, the Ministry of Health and Prevention (MOHAP) and Dubai Health Authority operate import-permit pathways for unregistered or restricted medicines on a named-patient basis. Saudi Arabia, Qatar, Kuwait, Bahrain, and Oman each operate analogous Ministry of Health named-patient frameworks with country-specific documentation requirements. India operates a personal-import pathway under the Drugs and Cosmetics Rules for quantities consistent with personal therapeutic use, typically up to three months supply per shipment. Turkey, Egypt, Jordan, Lebanon, and Morocco each operate distinct named-patient or import-permit pathways. Reserve Meds maintains current regulatory intelligence for each of these jurisdictions and prepares the documentation package required for each shipment.

What to expect through the coordination process

Patients beginning a request receive an initial coordinator contact within one business day of submission. The coordinator reviews the prescription, identifies the documentation required for the destination country, and provides an indicative cost range. Once required documents are uploaded - prescription, prescriber license verification, patient identification, and any indication-specific documentation such as genetic test results or biomarker reports - we issue a firm quote inclusive of product cost, packaging, shipping, regulatory documentation, and our coordination fee. Patients confirm acceptance of the firm quote before any sourcing or shipping is initiated, and Reserve Meds does not collect any deposit at intake. Once the firm quote is accepted and payment is received, the coordinator initiates sourcing through our licensed wholesale partners and prepares the destination-country regulatory package. Typical end-to-end timing from accepted quote to delivery is two to four weeks for ambient-shipped products and three to five weeks for cold-chain shipments, with variability driven by destination-country customs and regulatory turnaround times.

Not affiliated with the manufacturer

Reserve Meds is an independent operator of named patient program coordination. We are not a subsidiary, affiliate, authorised distributor, or licensed representative of any pharmaceutical manufacturer, and no statement on this page should be read as a partnership, endorsement, or representation by the named manufacturer. Trade names and product references on this page are used for informational purposes to identify the FDA-approved products that Reserve Meds coordinates through US specialty wholesale channels on behalf of named individual patients.

Privacy and data handling

Patient data submitted to Reserve Meds is handled under our HIPAA-aligned privacy policy and our destination-country data protection commitments. We minimise data collection to what is necessary for prescription verification, regulatory documentation, and shipping logistics. Patient identifiable information is shared with US wholesalers, destination-country regulatory authorities, and shipping carriers only as required to complete the coordination, and is not used for any marketing purpose.

Next step for patients

Patients or caregivers with a treating-physician prescription for an FDA-approved PTC product (Evrysdi or Emflaza) can begin a request now. Start a request ›

Next step for PTC teams

PTC Therapeutics medical affairs or commercial teams interested in cross-border NPP coordination for non-US territories can reach our BD team at [email protected].

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