How to access Empaveli from the UAE — the named-patient import pathway, 2026

By Reserve Meds · Clinical & regulatory team · Last reviewed 2026-04-23

A UAE patient with paroxysmal nocturnal hemoglobinuria (PNH) may receive a prescription for Empaveli (pegcetacoplan) from their treating haematologist. Empaveli is FDA-approved for PNH in adult patients, and it is manufactured by Apellis Pharmaceuticals. It is a first-in-class C3 complement inhibitor — a distinct mechanism from the C5 inhibitors Soliris and Ultomiris — designed to address the residual extravascular haemolysis that can persist for some PNH patients on C5 inhibitors alone. It is administered as a twice-weekly subcutaneous infusion. In the UAE, Empaveli may not yet be broadly registered, which is why your haematologist may be navigating a named-patient import pathway with you.

This guide explains the legal pathway, what documentation your physician needs, typical costs and timing, and where Reserve Meds fits in.

The clinical situation

Empaveli is administered as a 1,080 mg subcutaneous infusion twice weekly via a pump device, delivered over approximately 30 minutes per session. Because it inhibits complement upstream at the C3 level, it carries a US boxed warning regarding serious infections caused by encapsulated bacteria, which is why vaccination against Streptococcus pneumoniae, Neisseria meningitidis (types A, C, W, Y, and B), and Haemophilus influenzae type b (per current ACIP recommendations) is a gating step before therapy begins, as is enrolment in the US REMS program. Internationally, equivalent manufacturer risk-minimisation measures apply. Your treating haematologist confirms PNH diagnosis (flow cytometry), vaccination status, and the infusion plan per FDA labeling.

Is Empaveli legally importable into the UAE?

Yes — through the UAE Ministry of Health and Prevention (MoHAP) named-patient framework, with parallel authority through DoH Abu Dhabi and DHA Dubai depending on the prescribing facility. The pathway allows a UAE-licensed physician to import a medicine not locally registered when: (a) the medicine is approved by a recognised reference authority (FDA qualifies), (b) no locally available alternative is clinically equivalent for the patient, (c) the physician takes clinical responsibility, and (d) chain of custody is documented.

For pegcetacoplan specifically, international access respects the manufacturer's controlled-distribution program and the encapsulated-organism-infection risk-minimisation gating.

How the pathway works, step by step

1. Consultation with your treating haematologist. PNH diagnosis (flow cytometry), prior therapy history (especially any prior C5 inhibitor exposure and residual extravascular haemolysis evidence), and vaccination review.
2. Vaccination program. Pneumococcal, meningococcal (A/C/W/Y and B), and Hib vaccinations per current ACIP recommendations, completed at least 2 weeks before first dose where feasible.
3. MoHAP / DoH / DHA named-patient application. Your physician files the application.
4. US-side sourcing. Reserve Meds coordinates with our US-licensed specialty wholesale partner, aligned with the manufacturer's controlled-distribution model.
5. Cold-chain shipment. Empaveli ships at 2–8°C with continuous temperature monitoring.
6. Arrival and subcutaneous-pump training. The treating centre trains the patient or caregiver on the subcutaneous infusion pump; maintenance sessions are delivered at home twice weekly under physician supervision.

What documentation your physician needs

Your physician will typically need to provide:

• Clinical rationale letter confirming PNH diagnosis with flow-cytometry evidence, prior therapy history, and Empaveli as the indicated treatment
• Verification of UAE medical licence
• Patient identifier
• Vaccination documentation (pneumococcal, meningococcal A/C/W/Y and B, Hib)
• Planned twice-weekly infusion schedule and pump-training plan

Reserve Meds provides a physician documentation kit that bundles the templates MoHAP reviewers expect to see for complement inhibitors, including the encapsulated-organism vaccination block.

Costs and timing

Empaveli's US cash-pay reference price sits in a broad indicative range — the annualised treatment cost for PNH falls in the USD 450,000–600,000 range in US list pricing because of twice-weekly dosing. International cold-chain logistics, MoHAP documentation, and concierge coordination add incremental cost; Reserve Meds issues a full transparent quote at the start of intake. These ranges are indicative, not guaranteed.

Indicative timing — not guaranteed — for the first dose after cohort intake opens is approximately 14–28 days from the moment a complete application is submitted (vaccination lead time may extend this).

Reserve Meds is in pre-launch. Fulfillment availability is limited to our first cohort, and all timelines published on this site are indicative. If your clinical situation is time-sensitive, flag that when you join the waitlist — we triage accordingly.

Reserve Meds's role

Reserve Meds is a US-based concierge coordinator for cross-border specialty medicine. For Empaveli specifically, we provide:

• Sourcing. Through our US-licensed specialty wholesale partner, operating under DSCSA chain-of-custody and aligned with the manufacturer's controlled-distribution program.
• Documentation. Regulatory package for your physician and for MoHAP/DoH/DHA review, including the vaccination risk-minimisation block.
• Logistics. Cold-chain, temperature-monitored shipment; pump-consumables sourcing where applicable.
• Concierge case lead. A named point of contact.

What we do not do: We are not the prescriber. We do not practise medicine. We are not the dispensing pharmacy. All clinical decisions remain with your treating haematologist.

Frequently asked

Is this legal in the UAE? Yes, when executed through the MoHAP named-patient framework with appropriate documentation.

What about vaccinations? The encapsulated-organism-infection warning is load-bearing. Vaccination against meningococcus, pneumococcus, and Hib per current recommendations must be completed before starting Empaveli, with vaccinations ideally completed at least 2 weeks before first dose.

How does Empaveli compare with Soliris/Ultomiris? Soliris and Ultomiris inhibit C5 (terminal complement) and are administered IV. Empaveli inhibits C3 (upstream) and is administered as twice-weekly subcutaneous infusions via pump. Empaveli is particularly considered for patients on a C5 inhibitor with residual extravascular haemolysis (ongoing transfusion dependence despite complement inhibition). Your haematologist chooses based on haemolysis pattern and logistics.

Will private insurance cover this? Cash-pay is the default. Some UAE private insurers reimburse PNH therapy on escalated review; we supply documentation but do not process insurance claims directly.

Next step — join the first-cohort waitlist

Reserve Meds is opening to a limited first cohort in 2026. Add your case to the waitlist and our concierge case lead will reach out when we are ready to enter intake for Empaveli coordination in the UAE.

> Examples and timings above are composite illustrations drawn from published sources and typical named-patient patterns. Your individual case is assessed by your physician and our clinical-regulatory team; Reserve Meds does not guarantee outcomes or timelines.

Clinical & regulatory review: Reserve Meds clinical team and AI regulatory-counsel review pipeline. Last medically reviewed: 2026-04-23.