Factor XIII deficiency: cross-border specialty drug access for international patients

Quick orientation

Congenital Factor XIII deficiency is extremely rare with an estimated prevalence of 1 in 2 to 5 million people. Acquired forms are even rarer.

Typical age of onset. Congenital forms typically present in infancy with umbilical stump bleeding or intracranial hemorrhage.

Severity tiers. Untreated severe FXIII deficiency carries a high lifetime risk of intracranial hemorrhage. Prophylaxis prevents most bleeding.

Why specialty drugs for Factor XIII deficiency are hard to access internationally

FXIII concentrates and recombinant FXIII (catridecacog) are FDA-approved for prophylaxis but are not registered or widely available in many MENA and South Asian countries. The conditions are rare enough that local supply chains for these specific products often do not exist.

Treatments approved by the FDA

• Tretten (catridecacog, recombinant FXIII A-subunit) — FDA approval: 2013. Mechanism: Recombinant Factor XIII A-subunit. Route: Intravenous infusion monthly. US WAC ballpark: Approximately USD 25,000 to 35,000 per dose.
• Corifact (Factor XIII concentrate, plasma-derived) — FDA approval: 2011. Mechanism: Plasma-derived FXIII concentrate. Route: Intravenous infusion every 4 weeks. US WAC ballpark: Approximately USD 25,000 per dose.

Cross-border pathways used for Factor XIII deficiency

Most patients use one or more of the following regulatory pathways, depending on the destination country and the specific drug:

• Named Patient Program (NPP)
• Expanded Access Program (EAP)
• Compassionate Use
• Personal Import Scheme

What your physician needs to know

• Confirm subtype (FXIII A-subunit versus B-subunit deficiency) by genotyping.
• FXIII activity assay informs dosing.
• Lifelong prophylaxis is generally recommended for severe deficiency.
• Pediatric hematologist co-management is essential.
• Cold-chain or controlled-temperature shipping is required.

Common questions

How often is the infusion needed?

Typically every 4 weeks for prophylaxis.

Where can the infusion be administered?

At a hospital, outpatient clinic, or qualified home infusion service per local regulations.

Is the product available in my country?

Availability is limited globally. We confirm by destination.

Can I travel with my supply?

Patient travel with biologics requires destination-specific permits in many countries.

What documents are required?

Hematologist's prescription, FXIII activity result, genotype if known, and clinical summary.

Where Reserve Meds fits in

Reserve Meds is a cross-border specialty drug access platform. We support international patients whose prescribed FDA-approved medicine is not registered locally, is not reimbursed by their payer, or is otherwise unavailable through standard channels. For Factor XIII deficiency, our role is to coordinate the regulatory pathway, source the medicine from a DSCSA-compliant US wholesaler, and arrange validated cold-chain or controlled-temperature shipment to the destination country.

We do not replace your treating physician. We do not bill insurance. We operate a cash-pay model, and we work alongside the clinical team that knows your case. Every prescription is reviewed by a US-licensed pharmacist before dispense, and a US-licensed physician reviews the supply request before shipment.

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