Alnylam Pharmaceuticals: cross-border access for the specialty portfolio

Quick orientation

Alnylam Pharmaceuticals, Inc. is a US biotechnology company headquartered in Cambridge, Massachusetts, and is the global leader in RNA interference (RNAi) therapeutics. Alnylam has built the first commercial RNAi platform and holds the deepest pipeline of approved siRNA drugs. The company trades on NASDAQ as ALNY. FDA approval scale spans five marketed RNAi medicines across rare and common diseases, including hereditary transthyretin amyloidosis, acute hepatic porphyria, primary hyperoxaluria type 1, and elevated LDL cholesterol. Alnylam's commercial discipline is global: it operates direct subsidiaries in major markets and partners with Novartis on Leqvio for cardiovascular indications. The Alnylam pipeline continues to deepen across cardiometabolic, central nervous system, and rare-disease indications, with the platform's GalNAc-conjugate delivery technology enabling once-quarterly or semiannual subcutaneous dosing for hepatic targets.

Alnylam portfolio in cross-border NPP scope

Amvuttra (vutrisiran) is an FDA-approved subcutaneous RNAi therapy for hereditary transthyretin-mediated (hATTR) amyloidosis polyneuropathy in adults (FDA approval June 2022), with an expanded indication for ATTR cardiomyopathy approved in March 2025 based on HELIOS-B Phase 3 data. Amvuttra is dosed once every three months by subcutaneous injection. US list pricing is approximately US$463,000 per year. The product requires refrigerated cold-chain handling at 2 to 8 degrees C.

Oxlumo (lumasiran) is FDA-approved for primary hyperoxaluria type 1 (PH1) - an ultra-rare hereditary disease in which the liver overproduces oxalate, causing kidney stones and ultimately renal failure. Approved November 2020. Subcutaneous administration; weight-based dosing in pediatric and adult patients.

Onpattro (patisiran) was Alnylam's first commercial RNAi for hATTR polyneuropathy (IV infusion); it remains FDA-approved although Amvuttra is the more recent subcutaneous successor with a lighter administration burden.

Givlaari (givosiran) is FDA-approved for adults and adolescents with acute hepatic porphyria - an ultra-rare disease characterized by attacks of severe abdominal pain and neurological symptoms.

Leqvio (inclisiran), co-developed with Novartis and commercialised by Novartis, is an FDA-approved RNAi therapy for adults with primary hypercholesterolemia or heterozygous familial hypercholesterolemia, dosed every six months by subcutaneous injection.

Why patients route through cross-border NPP

Three structural drivers send Alnylam patients into cross-border NPP channels. First, the rare-disease indications - hATTR amyloidosis, primary hyperoxaluria, acute hepatic porphyria - have patient populations measured in hundreds per country, which means most national reimbursement systems either decline to list the products or require multi-year health technology assessments. Second, the ATTR cardiomyopathy expanded indication (Amvuttra March 2025) is substantially newer than the polyneuropathy indication, and most non-US jurisdictions have not yet aligned label coverage. Third, transthyretin cardiomyopathy in particular is now understood to affect a much larger population than historically diagnosed (especially men over 70 with heart failure and preserved ejection fraction), and confirmed-diagnosis patients in the GCC, India, Turkey, and Latin America frequently have no local pathway.

Pricing concentration also matters. At roughly US$463,000 per year list price, Amvuttra is well above typical out-of-pocket capacity, but informed cross-border families with private capacity routinely structure self-pay or part-pay arrangements where local reimbursement is unavailable. Patient profile is typically: confirmed transthyretin variant by genetic testing or wild-type ATTR confirmed by cardiac scintigraphy or biopsy; preserved performance status; and family or self-pay capacity for quarterly dosing.

Common cross-border destinations

The highest-frequency NPP destinations for Alnylam products from US-approved inventory are the United Arab Emirates, Saudi Arabia, India, Qatar, and Turkey. Major amyloidosis centers in Mumbai, Bangalore, Dubai, Abu Dhabi, Riyadh, and Istanbul drive most inbound referrals. The rare-disease specialty centers in these jurisdictions have built capacity for the diagnostic workup but face supply-side gaps for the targeted therapy.

Manufacturer engagement posture

Reserve Meds coordinates Alnylam-sourced US commercial product through DSCSA-compliant US specialty distributors. We are not affiliated with, owned by, or contracted as a distributor of Alnylam Pharmaceuticals. Our role is patient-facing coordination on behalf of named individual patients with a verified prescription from a treating physician. For Alnylam medical affairs, patient access, or commercial teams interested in cross-border NPP pathways, our BD contact is [email protected].

What Reserve Meds provides

For each Alnylam product request, Reserve Meds coordinates: prescription verification with the treating physician (typically a cardiologist, neurologist, or hepatologist depending on indication); DSCSA serialized chain-of-custody from Alnylam through licensed US wholesalers to the named patient; refrigerated cold-chain shipping (2 to 8 degrees C) with temperature data-loggers; 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 confirmed Alnylam-indicated diagnosis and a treating-physician prescription can begin a request now. Start an Amvuttra request › Start an Oxlumo request ›

For Onpattro, Givlaari, or Leqvio, please use our general request form and note the product name.

Next step for Alnylam teams

Alnylam medical affairs, access, or commercial teams interested in coordinating cross-border NPP for non-US territories can reach our BD team at [email protected].

Reserved for you.