Galsulfase (Intravenous)

Galsulfase is a treatment for the rare genetic condition Maroteaux-Lamy syndrome (MLS).

Overview

Galsulfase, sometimes known as Naglazyme, is an artificial enzyme which is used to replace an enzyme which is deficient in patients suffering from Maroteaux-Lamy syndrome (MLS), a rare genetic condition. It is administered once a week, through an injection, and it is meant to help patients live a more active lifestyle than they would otherwise be able to.

Patients suffering from Maroteaux-Lamy syndrome experience a broad range of different symptoms, including neurological issues such as deafness, compressed nerves, clouded corneas and thickened dura. It is usually spotted and diagnosed early in life, with one of the first signs of the disease being a delay in children learning to walk. Galsulfase has been tested as part of an enzyme replacement therapy and it has proven to be successful in improving the growth and joint movement of patients.

Galsulfase is taken in the form of an injected infusion, which is usually administered once a week. The galsulfase is mixed into a solution, which is given over the course of a four-hour sitting once weekly.

A course of galsulfase costs about $365,000 per patient per year and is therefore one of the most expensive drugs in the world.

Galsulfase is not a cure for the condition, it is a treatment which is designed to help improve the symptoms and the patient's ability to lead an active lifestyle. In order to continue having the desired effect on the condition, galsulfase treatment will have to be continued for throughout the patient's life.

Through taking galsulfase, patients usually experience much-improved endurance for physical activity, being able to walk for longer distances. It also helps to improve the lung function and the breathing capacity of patients.

In order for galsulfase treatment to be at its most effective, it should be started in patients as early as possible. The medication has a cumulative effect, and therefore improvements in the condition of patients are usually seen over a long period of time, rather than immediately. Once treatment has started, it is very important for it to be sustained and for weekly infusions to be taken in order for the medication to be able to have its full effect.

N the majority of cases, galsulfase is administered in a hospital or clinic. Patients will travel to the ward or clinic for half a day once a week to receive their treatment. However, in some cases, galsulfase can be administered at the patient's home.

Conditions treated

  • MLS (Maroteaux-Lamy syndrome)

Type of medicine

  • Enzyme replacement therapy

Side Effects

Galsulfase is a strong medication and is therefore more likely than other medications to cause patients to experience side effects. It is important to remember that if a doctor has recommended and prescribed this course of treatment, it will be in spite of the side effects that the treatment might cause. They will only prescribe the medication if they believe that the benefits of the treatment outweigh any potential side effects.

As the drug is usually administered in a hospital or a clinic, patients will be monitored throughout their treatment each week to check for any abnormal responses to the drug. If patients start to feel unwell as a result of the treatment, they should let a member of medical staff know as soon as possible.

Galsulfase may cause the following side effects:

  • Rash
  • Headaches
  • Chest pains
  • Fever and/or chills
  • Dizziness
  • Confusion
  • Nausea
  • Vomiting
  • Slushes
  • Coughing
  • Joint pain
  • High blood pressure

These side effects are most likely to occur whilst the infusion of galsulfase is being taken and for a short time afterwards.

Less common side effects of galsulfase include:

  • Redness of the eyes
  • Abdominal pain
  • Stomach cramps
  • Ear pain
  • Itching of the eyes

These side effects can usually be managed with other treatments or medications, but patients should inform their doctor and/or pharmacist if the side effects are prolonged or start to get worse. It may be that your doctor decided to prescribe an additional treatment such as antihistamines or corticosteroids in order to prevent side effects from happening again during treatment with galsulfase.

In very rare cases, patients may experience more severe side effects whilst taking galsulfase. These may include:

  • Changes in vision
  • Loss of hearing
  • Back pain
  • Kidney problems (changes in the color or amount of urine produced)
  • Incontinence
  • Difficulty to move arms and/or legs

If patients experience any of the symptoms listed above after taking galsulfase, they should seek urgent medical attention.

As galsulfase is a simulation of a naturally occurring enzyme within the human body, allergic reactions to this form of treatment are rare. However, if patients notice any of the following symptoms after taking galsulfase, they should seek urgent medical attention and state that there is a possibility they are having an allergic reaction:

  • Rash
  • Severe dizziness
  • Difficulty breathing
  • Itchiness
  • Swollen face, tongue, throat and/or lips

Dosage

When prescribing a patient with galsulfase treatment, doctors will take into account a variety of different factors in order to come up with a dosage recommendation. Doctors may adjust the dosage of medication according to variations in the age, weight, general condition and the overall health of the patient. As the solution is given in a hospital setting, and is administered by qualified healthcare professionals, the dosage is not something that patients will usually have to worry about directly.

In most cases, with no other extenuating circumstances, galsulfase is usually administered in the following dosage for adults:

- 1mg/kilogram of body weight, to be administered intravenously once a week.

It is usually mixed with a sodium chloride solution, to create a total volume of 250ml of the medication. The dose is then administered through an IV drip, over the course of four hours. The first 2.5% of the volume of the solution is to be given over the first hour, and the remaining solution is then administered over the course of the next three hours. This advice and dosage recommendation can vary from patient-to-patient and is likely to change dramatically in the case of children and pediatric doses.

Patients should be aware that the dosage of galsulfase may change throughout treatment if doctors do not see the effects they were hoping for. As this is a treatment taken over the course of a patient's lifetime, there are many opportunities for the dosage to change.

Interactions

There are no known interactions with galsulfase. This does not mean that no other pharmaceuticals cause interactions with galsulfase, but rather that not enough research has been done in order to find out what those interactions are and what the implications might be.

As the research has yet to be finalized, it is important for patients to keep an up-to-date and accurate list of any of the medications of they are taking. This list should include any over-the-counter medications, dietary supplements and herbal remedies.

It is also important for patents to closely monitor their condition for changes which might result from possible interactions between medications.

Warnings

It is possible for interactions to occur between pharmaceuticals and other medical conditions. In the case of galsulfase, it is especially important for patients to make their doctor or pharmacist aware if any of the following statements are true:

- You have a history of heart failure or other heart conditions
- You have a history of problems with your lungs
- You have any allergies to types of medication
- You are allergic to any of the ingredients of galsulfase
- You are scheduled to have surgery or have recently undergone a surgical procedure
- You are pregnant or planning to become pregnant
- You are currently breastfeeding or are planning to breastfeed soon

It is not known whether galsulfase can pass into breast milk, so patients should consult with their doctor before breastfeeding whilst having treatment with galsulfase.

Storage

Galsulfase is usually only administered within a hospital setting, and patients will therefore not have to worry about storing the drug in their own homes.

Before the infusion of Galsulfase is mixed together, the vial of medication should be stored in the refrigerator. As with all medications, galsulfase should be stored in its original packaging as supplied by the pharmacy. It should be clearly labelled so as to be identified easily and not to be confused with any other forms of medication.

The medication should be kept cold but should not be frozen. It should be stored in its opaque outer packing so as to be protected from exposure to light.

If in doubt, patients should consult their pharmacist for more details on how best to store this medication.

Care should always be taken to dispose of medication in a safe and controlled manner. It should not be flushed down the toilet or disposed with the household waste. Contact your hospital, clinic or pharmacy about medicine take-back programs and the best way of safely disposing of your medication.

Galsulfase should be kept out of the reach of children or pets.

Summary

Galsulfase is a form of medication used in cutting-edge enzyme replacement therapy for patients with a very rare and severe genetic condition, Maroteaux-Lamy syndrome (MLS). Patients with this condition usually experience difficulty in walking long distances and climbing stairs. One of the benefits of galsulfase is that it has been proven to help patients with Maroteaux-Lamy syndrome (MLS) to walk for longer periods of time and to be able to climb stairs, where they would be unable to do so without this treatment.

Galsulfase is not a cure for the condition, but it is a treatment which causes life-changing differences to the way in which patients with Maroteaux-Lamy syndrome (MLS) are able to live their lives day-to-day.

It is almost always administered in hospital, and the treatment usually takes half a day to complete. Patients will have to return to the hospital or clinic providing the treatment once a week in order to benefit from the full effects of the enzyme replacement therapy.

This drug has been known to provide life-changing differences to the lives of patients suffering from an extremely rare genetic condition. It is an example of cutting-edge medical technology and as such, is one of the most expensive drugs available today.