• Keep this leaflet. You may need to read it again.
• If you have any further questions, ask your doctor or nurse.
• If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. See section 4.

1. What Xenpozyme is and what it is used for
2. What you need to know before you are given Xenpozyme
3. How Xenpozyme is given
4. Possible side effects
5. How to store Xenpozyme
6. Contents of the pack and other information

Xenpozyme contains an enzyme called olipudase alfa.

Xenpozyme is used to treat an inherited disorder called acid sphingomyelinase deficiency (ASMD). It is used in children and adults with ASMD types A/B or B to treat the signs and symptoms of ASMD not related to the brain.

Patients with ASMD lack a properly working version of the enzyme acid sphingomyelinase. This results in build-up of a substance called sphingomyelin, which damages organs such as spleen, liver, heart, lungs and blood. Olipudase alfa acts in the same way as the natural enzyme would, and so acts as a replacement, reducing the build-up of sphingomyelin in the organs and treating the signs and symptoms.

• If you have experienced life-threatening allergic (anaphylactic) reactions to olipudase alfa (see section ‘Warnings and precautions’ below) or any of the other ingredients of this medicine (listed in section 6).

You may have side effects called infusion‑associated reactions (IARs) that may be caused by the infusion (drip) of the medicine. They may occur while you are being given Xenpozyme or within 24 hours after the infusion.

They may include allergic reactions (see section 4) and symptoms such as headache, a raised, itchy rash (hives), fever, nausea, vomiting and itchy skin.

If you think you are having an IAR, tell your doctor straight away.

If you have a severe allergic reaction during your infusion your doctor will stop your infusion and provide appropriate medical treatment. Your doctor will make a judgement about the risks and benefits of giving you further doses of Xenpozyme.

If you have a mild or moderate IAR, your doctor or nurse may temporarily stop the infusion, lower the infusion rate, and/or reduce the dose.

Your doctor may also give (or have given) you other medicines to prevent or manage allergic reactions.

Your doctor will order blood tests to check how well your liver is working (by measuring levels of your liver enzymes) before starting the treatment, and then at regular intervals as the doses are adjusted (see section 3).

Tell your doctor or nurse if you are using, have recently used, or might use any other medicines.

If you are pregnant or breast-feeding, think you may be pregnant, or are planning to have a baby, ask your doctor or nurse for advice before using this medicine.

There is no experience with the use of Xenpozyme in pregnant women. Xenpozyme may be harmful to unborn children when taken by a woman during pregnancy. Xenpozyme should only be used during pregnancy if clearly necessary. Women who are able to become pregnant should use effective contraception during treatment and for 14 days after the last dose if Xenpozyme is discontinued.

It is not known whether Xenpozyme passes into human breast milk. Xenpozyme was detected in animal milk. Tell your doctor if you are breast-feeding or plan to do so. Your doctor will then help you decide whether to stop breast-feeding, or whether to stop taking Xenpozyme, considering the benefit of breast-feeding the baby and the benefit of Xenpozyme to the mother.

Xenpozyme may have a minor influence on the ability to drive and use machines because you may experience low blood pressure (which may make you feel faint).

This medicine contains 3.02 mg sodium (main component of cooking/table salt) in each vial. This is equivalent to 0.15% of the recommended maximum daily dietary intake of sodium for an adult or an adolescent and ≤ 0.38% of the maximum acceptable daily intake of sodium for children below 16 years of age.

Tell your doctor immediately, if you suspect a change from your routine infusion. Because Xenpozyme will be given by a healthcare professional, an overdose is not likely.

It is important to have your infusion every 2 weeks. An infusion is considered missed if not given within 3 days from the scheduled infusion. Depending on the number of missed doses, your doctor may have to restart from a lower dose.

If you have missed an infusion or are unable to attend a scheduled appointment, please contact your doctor right away.

If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the Yellow Card Scheme at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store.

By reporting side effects you can help provide more information on the safety of this medicine.

• The active substance is olipudase alfa. One vial contains 20 mg of olipudase alfa.
• Other ingredients are
  
  • L-methionine
  • Sodium phosphate dibasic heptahydrate
  • Sodium phosphate monobasic monohydrate
  • Sucrose
  see section 2 Xenpozyme contains sodium

Xenpozyme is a powder for concentrate for solution for infusion in a vial (20 mg/vial).

The powder is white to off-white lyophilised powder. After mixing with sterile water, it is a clear, colorless solution. The solution must be further diluted before infusion.