Progressive familial intrahepatic cholestasis (PFIC): cross-border specialty drug access for international patients

Quick orientation

PFIC subtypes collectively affect an estimated 1 in 50,000 to 100,000 live births.

Typical age of onset. Most subtypes present in infancy or early childhood.

Severity tiers. Severity ranges substantially by subtype. PFIC1, PFIC2, and PFIC3 differ in genetic basis and clinical course. Severe forms progress to cirrhosis and may require transplant.

Why specialty drugs for Progressive familial intrahepatic cholestasis (PFIC) are hard to access internationally

Bylvay (odevixibat) and Livmarli (maralixibat) are IBAT inhibitors approved for cholestatic pruritus in PFIC and Alagille syndrome. International registration varies and pediatric supply continuity is essential.

Treatments approved by the FDA

• Bylvay (odevixibat) — FDA approval: 2021. Mechanism: Ileal bile acid transporter (IBAT) inhibitor. Route: Oral once daily. US WAC ballpark: Approximately USD 350,000 per year, weight-based. Country pricing: India · Oman · Lebanon.
• Livmarli (maralixibat) — FDA approval: 2021. Mechanism: Ileal bile acid transporter inhibitor. Route: Oral once daily. US WAC ballpark: Approximately USD 400,000 per year, weight-based. Country pricing: UAE · Saudi Arabia · India · Qatar · Kuwait · Jordan · Lebanon.

Cross-border pathways used for Progressive familial intrahepatic cholestasis (PFIC)

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
• Rare Pediatric Disease

What your physician needs to know

• Confirm PFIC subtype by genotyping.
• Document pruritus severity and serum bile acid levels.
• Pediatric hepatology co-management is essential.
• Monitor fat-soluble vitamin levels.
• Liver function monitoring and growth tracking.

Common questions

How do Bylvay and Livmarli compare?

Both are IBAT inhibitors with similar mechanisms. Selection depends on age range, formulation preference, and clinical factors.

Are they available outside the US?

Registration is rolling out. We confirm by destination.

Will they replace liver transplant?

Some patients see significant improvement; others may still require transplant. Long-term data continues.

How is the dose adjusted?

Weight-based dosing; updates with each refill as the child grows.

What documents are required?

Pediatric hepatologist's prescription, genetic confirmation, 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 Progressive familial intrahepatic cholestasis (PFIC), 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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