Congenital adrenal hyperplasia (CAH): cross-border specialty drug access for international patients

Quick orientation

Classic CAH affects approximately 1 in 15,000 live births worldwide. Non-classic forms are substantially more common, particularly in some ethnic populations including Ashkenazi Jewish, Hispanic, and certain Mediterranean groups where carrier frequency can exceed 1 in 30.

Typical age of onset. Classic forms present at birth or in early infancy; non-classic forms in adolescence or adulthood.

Severity tiers. Classic CAH ranges from salt-wasting (most severe) to simple virilizing. Untreated salt-wasting CAH is life-threatening in the neonatal period.

Why specialty drugs for Congenital adrenal hyperplasia (CAH) are hard to access internationally

Crenessity (crinecerfont), approved by the FDA in 2024, is the first targeted therapy for classic CAH and aims to reduce supraphysiologic glucocorticoid exposure that drives long-term morbidity. Registration in most international markets is in early stages.

Treatments approved by the FDA

• Crenessity (crinecerfont) — FDA approval: 2024. Mechanism: Corticotropin-releasing factor type 1 receptor (CRF1) antagonist. Route: Oral twice daily. US WAC ballpark: Approximately USD 150,000 to 180,000 per year. Country pricing: UAE · India · Qatar · Kuwait · Jordan · Lebanon.

Cross-border pathways used for Congenital adrenal hyperplasia (CAH)

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 21-hydroxylase deficiency genotype and current glucocorticoid regimen.
• Document inability to reduce glucocorticoid dose without androgen excess on standard care.
• Crenessity is added to glucocorticoid therapy, not a replacement.
• Bone age, growth velocity, and androgen levels inform monitoring.
• Pediatric endocrinologist co-management is recommended.

Common questions

Will Crenessity replace my hydrocortisone?

No. It is added to glucocorticoid therapy and allows a reduction in dose, not discontinuation.

Is Crenessity approved for children?

The FDA label includes patients aged 4 years and older.

What about stress-dose steroids?

Standard stress dosing still applies during illness, surgery, or trauma. Your endocrinologist provides the protocol.

Is it available internationally?

Registration is in early stages outside the US. We confirm current status by destination.

What documents are required?

Genetic confirmation, current regimen, recent androgen panels, and treating endocrinologist's prescription.

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 Congenital adrenal hyperplasia (CAH), 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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