What Xenpozyme is
Xenpozyme contains an enzyme called olipudase alfa.
What Xenpozyme is used for
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.
How Xenpozyme works
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.

You must not be given Xenpozyme
- 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).
Warnings and precautions

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 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).

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

Pregnancy and breast-feeding
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 contraception while using Xenpozyme.
It is not known whether Xenpozyme passes into breast 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.

Driving and using machines
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).

Xenpozyme contains sodium
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.

Xenpozyme will be given to you as a drip (infusion) under the supervision of a healthcare professional who is experienced in the treatment of ASMD or other metabolic diseases.
The dose you receive is based on your body weight and will be given to you every two weeks. Treatment starts with a low dose of the medicine, which is gradually increased.
Infusion usually lasts around 3 to 4 hours but may be shorter or longer based on your doctor’s judgement, and may be shorter during the period whilst your dose is being increased.
Adult patients
The recommended starting dose of Xenpozyme is 0.1 mg for each kg of body weight. This is increased in a planned way with each subsequent dose, until the recommended dose of 3 mg for each kg of body weight every 2 weeks is reached. It typically takes up to 14 weeks to reach the recommended dose but may be longer based on your doctor’s judgement.
Children
The recommended starting dose of Xenpozyme is 0.03 mg for each kg of body weight. The subsequent doses should be increased in a planned way up to the recommended dose of 3 mg for each kg of body weight every 2 weeks. It typically takes up to 16 weeks to reach the recommended dose but may be longer based on your doctor’s judgement.
Home infusion
Your doctor may consider home infusion of Xenpozyme if you are on stable dose and tolerating your infusions well. This decision to move to home infusion should be made after evaluation and recommendation by your doctor. If you get a side effect during an infusion of Xenpozyme, the person giving your home infusion may stop the infusion and start appropriate medical treatment.
Instructions for proper use
Xenpozyme is given by intravenous infusion (a drip into a vein). It is supplied as a powder that will be mixed with sterile water before it is given.
If you are given more Xenpozyme than you should
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.
If you miss a Xenpozyme infusion
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 have any further questions on the use of this medicine, ask your doctor or nurse.

Like all medicines, this medicine can cause side effects, although not everybody gets them.
Infusion-associated reactions (IARs) have been seen while patients were being given the medicine or within 24 hours after the infusion.

The most serious side effects may include sudden severe allergic reactions, raised, itchy rash (hives), rash, increased liver enzymes and irregular heartbeat.
You must tell your doctor immediately if you get an IAR or an allergic reaction.
If you have an infusion reaction you may be given additional medicines to treat or help prevent future reactions. If the infusion reaction is severe, your doctor may stop the infusion of Xenpozyme and start giving appropriate medical treatment.

Very common (may affect more than 1 in 10 people):

• Headache
• Fever – body temperature increased
• Raised, itchy rash (hives)
• Nausea
•  Vomiting
• Abdominal (belly) pain
• Muscles aches
• Itchy skin
• Increased blood test for inflammation

Common (may affect up to 1 in 10 people):

• Rash (different types of rash sometimes with itch)
• Pain in upper belly
• Fatigue
• Abnormal blood test for liver function
•  Diarrhoea
•  Reddening of the skin
• Joint pain
• Back pain
• Chills
• Difficulty breathing
• Abdominal discomfort
• Bone pain
• Pain
• Low blood pressure
• Forceful heartbeat that may be rapid or irregular
• Fast heartbeat
• Liver pain
• Severe allergic reactions
• Feeling very warm
• Throat and voice box irritation
• Throat tightness and swelling
• Wheezing
• Skin lesions (such as solid elevated or red flat lesions) • Rapid swelling under the skin in areas such as the face, throat, arms and legs which can be life threatening if throat swelling blocks the airway
• Stomach pain
• Itchy or red eyes
• Eye discomfort
• Weakness
• Abnormal blood test for inflammation
• Catheter site-related reactions including pain, itching, or swelling

Reporting of side effects

• Saudi Arabia

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Keep this medicine out of the sight and reach of children.
Do not use Xenpozyme after the expiry date stated on the label and the carton. The expiry date refers to the last day of the month.
Store in refrigerator between 2°C to 8°C.
After dilution, immediate use is recommended.
If not used immediately, the reconstituted solution may be stored for up to 24 hours at 2°C to 8°C or up to 12 hours at room temperature (up to 25°C).
After dilution, the solution can be stored for up to 24 hours at 2-8°C followed by 12 hours (including infusion time) at room temperature.
Do not throw away any medicines via wastewater or household waste. Ask your doctor or nurse how to throw away medicines you no longer use. These measures will help protect the environment.