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Niemann-Pick disease (ASMD and type C): 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.

Niemann-Pick disease: the short answer

The US FDA has approved three medicines on this page for Niemann-Pick disease, each for specific patients. Two 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: Xenpozyme, Miplyffa and Aqneursa.
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 Niemann-Pick disease?

Niemann-Pick disease is an inherited condition that affects many body systems. It has four main types: A, B, C1 and C2. Types A and B are caused by a shortage of the enzyme acid sphingomyelinase and are now often called acid sphingomyelinase deficiency (ASMD).^{[1, 2]}

Infants with type A usually develop an enlarged liver and spleen by 3 months, fail to grow as expected, and lose skills from about age 1, with lung damage and a cherry-red spot in the eye. Type B is milder and usually appears in mid-childhood with an enlarged liver and spleen, lung infections and low platelets. Most people with type B live into adulthood.^[1]

Types C1 and C2 differ only in their genetic cause and usually appear in childhood with neurological problems. In the US, an enzyme replacement therapy is approved for the non-brain features of ASMD, and two medicines are approved for the neurological features of type C.^{[1, 3, 4, 5]}

Niemann-Pick disease in the Gulf

A 1990 review of lysosomal storage diseases referred in Saudi Arabia found that Niemann-Pick disease type B was among the conditions seen more often than elsewhere, likely linked to marriages within families.^[6]

A 2024 case report described a Saudi child with type A confirmed by genetic testing of the SMPD1 gene.^[2]

US FDA-approved medicines for Niemann-Pick disease

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.

Xenpozyme (olipudase alfa-rpcp)
FDA-approved for non-central nervous system features of acid sphingomyelinase deficiency (ASMD, Niemann-Pick types A and B) in adults and children. It is an enzyme replacement therapy 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.^{[7, 8]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman
Miplyffa (arimoclomol citrate)
FDA-approved for neurological features of Niemann-Pick disease type C in adults and children 2 years and older, together with miglustat. It is a capsule.^[4]
Gulf registers: Saudi Arabia: not found by brand name in the SFDA drugs list. Abu Dhabi: not found by brand name 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.^{[7, 8]}
Country guides: Turkey
Aqneursa (levacetylleucine)
FDA-approved for neurological features of Niemann-Pick disease type C in adults and children weighing at least 15 kg. It is granules for oral suspension.^[5]
Gulf registers: Saudi Arabia: not found by brand name 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.^{[7, 8]}
Country guides: Saudi Arabia, UAE, Bahrain, Egypt, Jordan, Turkey, 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.^{[9, 10]} 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 Niemann-Pick disease

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 Niemann-Pick disease?

Yes. The medicines on this page have US FDA approval for specific groups of patients: Xenpozyme, Miplyffa and Aqneursa. The exact conditions are in each US label and are summarized above. Your specialist decides whether any of them fits.

Is Xenpozyme 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 Xenpozyme?

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.

Does the enzyme therapy treat brain symptoms?

No. The US label for Xenpozyme covers the non-central nervous system features of ASMD. The two type C medicines on this page are for neurological features of type C.

Which type does my child have?

The type is confirmed by enzyme and genetic tests. Your metabolic specialist explains the result and which medicine, if any, fits.

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.