Vimizim - Elosulfase alfa uses, dose and side effects

Vimizim contains an enzyme , elosulfase alfa, which belongs to a group of medicines called enzyme replacement therapies. It is used to treat adults and children with mucopolysaccharidosis type IVA (the disease MPS IVA, also called Morquio A syndrome).

People with the disease MPS IVA completely lack or do not have enough N-acetylgalactosamine-6-sulfatase, an enzyme that breaks down certain substances in the body such as keratan sulfate that is found in many tissues in the body, including cartilage and bone. As a result, these specific substances are not broken down and processed by the body as they should. They accumulate in the tissues , interfering with their normal function and causing the symptoms of MPS IVA, such as difficulty walking, difficulty breathing, short stature and hearing loss.

How Vimizim works

This drug replaces the natural enzyme N-acetylgalactosamine-6-sulfatase that is missing in MPS IVA patients. The treatment has been shown to improve walking ability and reduce the levels of keratan sulfate in the body. This medicine can improve the symptoms of MPS IVA.

What you need to know before you use Vimizim

Do not use Vimizim

If you have had a life-threatening allergic reaction to elosulfase alfa or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

If you are treated with Vimizim, you may experience infusion reactions. An infusion reaction is a side effect , including an allergic reaction , that occurs during the infusion or within 24 hours after the infusion (see section 4). If you experience such a reaction, contact your doctor immediately.
If you have an allergic reaction during infusion one, your doctor may slow down or stop infusion one. Your doctor may also give you additional medications to treat any allergic reactions (eg antihistamines and/or corticosteroids ( Cortisone )).
If you experience back pain, numbness in the arms or legs, or loss of control over urination or defecation, contact your doctor immediately. These problems may be part of the disease and may be due to pressure on the spinal cord.

Other medicines and Vimizim

Tell your doctor if you are taking, have recently taken or might take any other medicines.

Pregnancy, breastfeeding and fertility

You should not take Vimizim during pregnancy unless absolutely necessary. It is not known whether Vimizim is excreted in breast milk. Discuss with your doctor whether the benefits of taking Vimizim outweigh the possible risk to your newborn baby while breastfeeding. It is not known what effects Vimizim has on human fertility . No effect on fertility was observed in animals.

Driving ability and use of machinery

Dizziness has been reported in some patients during Vimizim infusion. Tell your doctor if you feel dizzy after the infusion , especially if you are going to drive or use machines where dizziness can be dangerous.

Vimizim contains sodium and sorbitol (E420)

This medicine contains 8 mg sodium (main ingredient in table salt) per 5 ml vial . This corresponds to 0.4% of the recommended daily dietary intake of sodium for an adult.

This medicine contains 100 mg of sorbitol per 5 ml vial , which corresponds to 40 mg/kg. Sorbitol is a source of fructose. If you (or your child) have hereditary fructose intolerance (HFI), a rare genetic disorder, you (or your child) must not take this medicine unless you have discussed it with a doctor. Patients with HFI cannot break down fructose and this can cause serious side effects . Before taking this medicine, you must tell the doctor if you (or your child) have HFI or if your child stops eating candy and sweets or drinks sugary drinks because they feel sick, are vomiting or experience unpleasant effects such as a swollen stomach, stomach cramps or diarrhea.

How to use Vimizim

Your doctor or nurse will give Vimizim to you by infusion into a vein.

The medicine must be diluted before it is given. Your doctor or nurse will give you some medicines before treatment to reduce allergic reactions and you may also be given medicines to treat any fever.

Dose

Dose one is based on your body weight. The recommended dosage for adults and children is 2 mg/kg body weight given once a week as a drip ( intravenous infusion ) and it is the same for adults and children. Each infusion takes about 4 hours. Treatment with Vimizim can be started at the earliest possible age, and is intended for long-term treatment.

If you have any further questions about this medicine, ask your doctor or nurse.

Possible side effects

Like all medicines, this medicine can cause side effects , although not everybody gets them.

Side effects were mainly reported in connection with the patients receiving the drug or shortly afterwards (“infusion reactions”). The most serious side effects were serious allergic reactions (which are uncommon – may affect up to 1 in 100 people) and mild and moderate vomiting (which are very common – may affect more than 1 in 10 people). Symptoms of an allergic reaction include rash, itching or hives on the skin (which are common – may affect up to 1 in 10 people). If you experience difficulty swallowing, severe shortness of breath or wheezing, swelling of the face or lips, dizziness or weak pulse , these may be symptoms of a seriousallergic reaction and you should tell your doctor immediately . Based on the severity of the side effect , your doctor may slow down or temporarily stop the infusion and/or give you additional medicines to reduce the effects of a severe allergic reaction (eg antihistamines and/or corticosteroids ) or to reduce fever. (antipyretic).

Very common side effects include symptoms of infusion reactions such as headache, nausea, fever, chills and stomach ache. Other very common side effects are diarrhoea, pain in the mouth and throat, dizziness and difficulty breathing.

Common side effects observed were muscle pain.

How to store Vimizim

Keep this medicine out of the sight and reach of children.

Use before the expiry date stated on the carton and on the vial after Ex.date. The expiration date is the last day of the specified month.

Unopened vials:

Store in a refrigerator (2°C–8°C).

Do not freeze.

Store in the original packaging. Light sensitive.

Do not use Vimizim if the solution is discolored or contains visible particles.

After dilution:

The diluted solution should be used immediately. If not used immediately, in-use storage times and conditions are the responsibility of the user and would normally not be longer than 24 hours at 2°C–8°C followed by up to 24 hours at 23°C–27°C during infusion .

Medicines must not be thrown into the drain or among household waste. Ask the pharmacist how to dispose of medicines that are no longer used. These measures will help to protect the environment.

Contents of the packaging and other information

Contents declaration

The active substance is elosulfase alfa. Each ml of concentrate contains 1 mg of elosulfase alfa. Each 5 ml vial contains 5 mg elosulfase alfa.
Other ingredients are: sodium acetate trihydrate, monosodium phosphate monohydrate, arginine hydrochloride, sorbitol, polysorbate 20 and water for injections (see section 2 under “Vimizim contains sodium and sorbitol (E420)”).

Appearance and package sizes of Vimizim

Vimizim is supplied as concentrate for infusion , solution (sterile concentrate). The clear to slightly opalescent and colorless to pale yellow concentrate should be free of visible particles.

Package sizes: 1 vial of 5 ml.

Marketing Authorisation Holder

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork, P43 R298

Ireland

Manufacturer