Alexion: cross-border access for the specialty portfolio

Quick orientation

Alexion was acquired by AstraZeneca in July 2021 and operates as Alexion, AstraZeneca Rare Disease, based in Boston, Massachusetts. The Alexion specialty footprint is exclusively in rare and ultra-rare disease, with the complement-pathway franchise as the centerpiece (Soliris, Ultomiris, Voydeya in PNH; the metabolic rare-disease products Strensiq for hypophosphatasia and Kanuma for lysosomal acid lipase deficiency; Andembry, the kallikrein inhibitor for hereditary angioedema; Koselugo, the MEK inhibitor for neurofibromatosis type 1 plexiform neurofibromas, co-marketed with Merck through a Merck partnership predating the AZ acquisition).

For Reserve Meds cross-border named-patient programs, the Alexion portfolio is the single highest concentration of products where local registration is the exception rather than the rule. Patients on these therapies in MENA and India most often route cross-border by default.

Portfolio in cross-border NPP scope

Soliris (eculizumab)

Terminal complement C5 inhibitor approved for paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis (anti-AChR positive), and anti-AQP4-positive neuromyelitis optica spectrum disorder. US WAC at the standard maintenance every-two-weeks schedule has historically run at approximately $700,000 per patient per year. Cross-border requests come from PNH, aHUS, gMG, and NMOSD patients in MENA and India where local registration may cover only one or two of these four indications.

Ultomiris (ravulizumab)

Long-acting terminal complement C5 inhibitor approved for PNH, aHUS, gMG, and NMOSD. Ultomiris is the once-every-eight-weeks successor to Soliris (which dosed every two weeks). Cross-border requests favor Ultomiris for the dosing interval, but transition planning must account for the weight-based loading and maintenance schedule. International requests cluster around adult PNH patients seeking to move from Soliris to the longer-interval regimen.

Voydeya (danicopan)

Oral factor D complement inhibitor approved as an add-on to a C5 inhibitor (Soliris or Ultomiris) for paroxysmal nocturnal hemoglobinuria with extravascular hemolysis. International requests come from PNH patients on Soliris or Ultomiris who continue to experience extravascular hemolysis and where Voydeya is not yet locally registered.

Strensiq (asfotase alfa)

Recombinant alkaline phosphatase enzyme replacement therapy approved for perinatal-, infantile-, and juvenile-onset hypophosphatasia. International requests come from pediatric hypophosphatasia patients in MENA and India where the disease is recognized and ERT is the only disease-modifying option but local product is unavailable.

Kanuma (sebelipase alfa)

Recombinant lysosomal acid lipase enzyme replacement therapy for LAL deficiency (Wolman disease and cholesteryl ester storage disease). Cross-border requests are by definition very rare but very high acuity: LAL deficiency is ultra-rare and Kanuma is the only on-label option.

Andembry (garadacimab)

Factor XIIa inhibitor monoclonal antibody approved 2025 for prophylaxis of hereditary angioedema attacks in adults and adolescents. International requests come from HAE patients in MENA and India where Andembry's monthly subcutaneous dosing has not yet been added to local labels.

Koselugo (selumetinib, co-marketed with Merck)

MEK inhibitor approved for pediatric patients age 2 and older with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. International requests come from pediatric NF1 patients in MENA and India where the locally registered options for inoperable plexiform neurofibromas are essentially supportive care only.

Andexxa (andexanet alfa)

Recombinant factor Xa decoy approved for reversal of anticoagulation from apixaban or rivaroxaban in life-threatening or uncontrolled bleeding. International requests are rare but acute and emergency-driven.

Why patients route Alexion products through cross-border NPP

The Alexion cross-border driver is structural: ultra-rare disease products by definition serve patient populations too small to justify local commercial registration in many MENA and India markets. The complement-pathway franchise (PNH, aHUS, gMG, NMOSD), the bone-rare-disease franchise (HPP, LAL deficiency), and the NF1 pediatric franchise are all populations where cross-border NPP is the default access route, not the exception.

Alexion OneSource is the US-resident-only patient assistance program. International patients route as cash-pay through the named-patient pathway.

Manufacturer engagement posture

Reserve Meds coordinates Alexion-manufactured US product through DSCSA-compliant specialty wholesaler channels. We are not an authorized Alexion distributor; we are an independent named-patient program coordinator. Patient assistance program coverage does not extend to cross-border patients. All Reserve Meds orders are cash-pay at firm-quote pricing.

For Alexion access, medical affairs, and international new product planning teams: Reserve Meds maintains structured demand visibility across the complement-pathway, metabolic rare-disease, HAE, and NF1 franchises. If your group is sizing pre-registration or cross-border supplemental demand in MENA and India, we are open to a structured conversation under NDA.

Common cross-border destinations for the Alexion portfolio

The complement franchise (Soliris, Ultomiris, Voydeya) concentrates in tertiary hematology and renal centers in the UAE, Saudi Arabia, India, Egypt, and Kuwait. Strensiq and the bone-rare-disease franchise serve specialty metabolic centers across MENA and India. Andembry tracks HAE specialty allergy and immunology centers. Koselugo serves pediatric neurology and oncology centers in the Gulf and India.

What Reserve Meds provides

For every Alexion product order, Reserve Meds coordinates DSCSA chain-of-custody documentation; cold-chain validated logistics (all Alexion products require 2-8 degree C storage); 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 an Alexion product and want a firm quote can start a request below.

Start a request for an Alexion product

Next step for Alexion teams

If you are on the Alexion or AstraZeneca Rare Disease 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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