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Paroxysmal nocturnal hemoglobinuria (PNH): US-approved treatments and how Gulf families can access them

Creator: Reserve Meds
Published: 2026-10-17

A plain guide for families in the Gulf, checked against US FDA labels, Gulf drug registers and published medical research.

Reviewed by the Reserve Meds clinical operations team on 17 October 2026.

Paroxysmal nocturnal hemoglobinuria: the short answer

The US FDA has approved six medicines on this page for paroxysmal nocturnal hemoglobinuria, each for specific patients. Six of them are on at least one Gulf register we checked, so ask your hospital about local supply first. Otherwise the hospital can request a named-patient import. Reserve Meds is a digital health company. We do not diagnose or prescribe.

US FDA-approved medicines on this page: Soliris, Ultomiris, Empaveli, Fabhalta, Piasky and Voydeya.
Gulf registration (checked 17 October 2026): see each medicine below.
Legal route: Local supply where listed; otherwise a named-patient import request filed by the treating hospital.
Price: Quote on request.

What is paroxysmal nocturnal hemoglobinuria?

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, not inherited, disorder that causes early destruction and poor production of blood cells. It can occur at any age but is most often diagnosed in young adults.^[1]

People with PNH have episodes in which red blood cells break down early (hemolysis), sometimes passing dark urine, often noticed in the morning. This causes anemia with tiredness, weakness, pale skin and shortness of breath. PNH also raises the risk of blood clots, especially in large abdominal veins, and of infections.^[1]

PNH is linked with complement, part of the immune system that attacks the abnormal blood cells. In the US, several complement inhibitors are approved, given as infusions, injections or tablets.^{[2, 3, 4]}

Paroxysmal nocturnal hemoglobinuria in the Gulf

A 2025 review of PNH in the Middle East found no regional registry data and only 15 relevant publications, five of them from Saudi Arabia. An Omani cohort estimated an incidence of 1.9 per 5 million people.^[2]

US FDA-approved medicines for paroxysmal nocturnal hemoglobinuria

Each medicine below is approved in the US for a specific group of patients. The wording summarizes the current US label. Your specialist decides whether any of them is right for the patient.

Soliris (eculizumab)
FDA-approved for patients with PNH, to reduce hemolysis. It is given by intravenous infusion.^[3]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, India, Pakistan
Ultomiris (ravulizumab)
FDA-approved for adults and children one month and older with PNH. It is given by intravenous infusion every 8 weeks for adults after loading doses.^[7]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, India
Empaveli (pegcetacoplan)
FDA-approved for adults with PNH. It is an infusion under the skin.^[8]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, India
Fabhalta (iptacopan)
FDA-approved for adults with PNH. It is a capsule taken by mouth.^[4]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, India, Pakistan
Piasky (crovalimab)
FDA-approved for adults and children 13 years and older with PNH who weigh at least 40 kg. It is given by injection.^[9]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Voydeya (danicopan)
FDA-approved for add-on therapy to ravulizumab or eculizumab for extravascular hemolysis in adults with PNH. It is a tablet.^[10]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[5, 6]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, India

How families in the Gulf can access these medicines

If the medicine is listed in your country. Ask your specialist and the hospital pharmacy about local supply first. A listing does not mean every hospital stocks the medicine, and another brand of the same medicine may also be registered, but local supply is usually the fastest route and you may not need us.

If it is not registered. The usual legal route is a named-patient request: the treating hospital, on your specialist's written request, asks the national regulator for permission to import the medicine for one patient, using the US FDA approval as the reference. In Saudi Arabia the SFDA Special Access Program guidance covers these requests; in Bahrain, NHRA Circular 41 of 2020 sets the procedure. Other Gulf countries have their own procedures, which the hospital pharmacy confirms.^{[11, 12]} Once approval is in place, Reserve Meds arranges supply through a US-licensed pharmaceutical wholesale partner to the hospital pharmacy.

Who does what

Your treating specialist decides whether a medicine is right for the patient and writes the letter.
The hospital checks local supply, files any import request, and gives the treatment safely.
The national regulator decides whether to approve the import of a medicine that is not registered.
The manufacturer decides where its medicine can be supplied, especially for cell and gene therapies.
Reserve Meds helps organize records, coordinates US supply through our wholesale partner, shipping and updates to your family. We do not give medical advice.

Documents you will usually need

A letter from the treating specialist with the diagnosis, past treatments and why the medicine is being considered
Reports that confirm the diagnosis, such as laboratory, genetic, imaging or pathology results, translated into English where needed
Recent clinic notes and a list of current medicines
For a hospital import request: the prescription, the hospital's request form and the written consent of the patient or guardian
Proof of the US FDA approval and the US prescribing information (we can supply these)

We give you a written timeline estimate once we see the hospital file. The regulator, the treatment center and the manufacturer set their own timelines.

What treatment costs

Quote on request. Your total cost depends on the medicine, the dose, shipping, import steps and coordination. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help your doctor and hospital prepare the file, arrange US supply and temperature-controlled shipping once approval is in place, and keep your family updated. We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them.

Ask us about treatment for paroxysmal nocturnal hemoglobinuria

Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.

Frequently asked questions

Is there an FDA-approved treatment for paroxysmal nocturnal hemoglobinuria?

Yes. The medicines on this page have US FDA approval for specific groups of patients: Soliris, Ultomiris, Empaveli, Fabhalta, Piasky and Voydeya. The exact conditions are in each US label and are summarized above. Your specialist decides whether any of them fits.

Is Soliris available in Saudi Arabia or the UAE?

Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register. A listing in Abu Dhabi does not show federal UAE status.

How can a family in the Gulf get Soliris?

Ask the treating hospital about local supply first. If it is not available, the hospital can file a named-patient import request with the national regulator on your specialist's written request. Once approved, we arrange US supply to the hospital pharmacy.

How much does treatment cost?

Quote on request. Your total cost, including the medicine, shipping, import steps and coordination, is quoted in writing for your case before you commit. No hidden charges.

Why do complement inhibitors need vaccines?

Medicines that block complement raise the risk of serious meningococcal infections. Their US labels require vaccination and a safety program before treatment. Your hematologist arranges this.

Is PNH inherited?

No. MedlinePlus Genetics describes PNH as an acquired disorder, not inherited.

Sources for the last two answers: ^{[3, 4]} ^[1]

Sources

Checked on 17 October 2026. Labels, registers and regulator rules change. Your hospital pharmacy confirms the current position before filing.

This page is general health information, not medical advice. It does not replace a consultation with your doctor.