Galsulfase

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You should not use this medication if you are allergic to galsulfase or mouse proteins.

Before receiving galsulfase, tell your doctor if you are allergic to any drugs, or if you have:

• sleep apnea (breathing stops during sleep);
• a fever; or
• flu symptoms, or a common cold.

If you have any of these conditions, you may need a dose adjustment or special tests to safely use galsulfase.

Your name may need to be listed on a patient registry while you are using this medication. The purpose of this registry is to track the progression of this disorder and the effects that galsulfase has on long-term treatment of Maroteaux-Lamy syndrome.

FDA pregnancy category B. This medication is not expected to be harmful to an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant during treatment.

It is not known whether galsulfase passes into breast milk or if it could harm a nursing baby. Do not receive this medication without telling your doctor if you are breast-feeding a baby.

Galsulfase is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis VI (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis type 6), or MPS VI, also called Maroteaux-Lamy syndrome.

MPS VI is a metabolic disorder in which the body lacks the enzyme needed to break down certain sugars and proteins. These substances can build up in the body, causing enlarged organs, abnormal bone structure, changes in facial features, breathing problems, heart problems, vision or hearing loss, and changes in mental or physical abilities.

Galsulfase may improve walking and stair-climbing ability in people with this condition. However, this medication is not a cure for MPS VI.

Galsulfase may also be used for purposes not listed in this medication guide.

Galsulfase is injected into a vein through an IV. You will most likely receive this injection in a clinic or hospital setting.

Galsulfase must be given slowly through an IV infusion, and can take at least 4 hours to complete.

Your doctor may also prescribe other medications to help prevent an allergic reaction to galsulfase. Take all of your medications as directed.

Galsulfase is usually given once per week. Follow your doctor's instructions.

Your doctor will need to check your progress while you are using galsulfase.

Usual Adult Dose for Mucopolysaccharidosis Type VI:

1 mg/kg IV once a week

Comments:
-Clinical studies did not include patients older than 29 years and it is therefore unknown if they respond differently than younger patients.

Use: For the treatment of patients with Mucopolysaccharidosis VI (MPS VI [Maroteaux-Lamy syndrome])

Usual Pediatric Dose for Mucopolysaccharidosis Type VI:

5 years or older: 1 mg/kg IV once a week

Use: For the treatment of patients with MPS VI (Maroteaux-Lamy syndrome)

• It is used to treat mucopolysaccharidosis VI.

Use galsulfase as ordered by your doctor. Read all information given to you. Follow all instructions closely.

• It is given as an infusion into a vein over a period of time.
• Other drugs may be given before this medicine to help avoid side effects.

What do I do if I miss a dose?

• Call your doctor to find out what to do.

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

After calculating dose, round to the nearest whole vial to prepare infusion. Dilute in NS to a final volume of 250 mL (including volume of galsulfase). Slowly add galsulfase to infusion bag (use a low-protein binding container; compatibility in glass containers has not been studied). For patients ≤20 kg or with fluid restriction, the dose may be added to 100 mL NS (total volume is 100 mL is not necessary). Gently rotate bag to distribute. Do not shake or agitate, do not use filter needle.

There are no known significant interactions.

Note: Percentages reported are from a placebo-controlled study (39 patients, 19 on galsulfase); also included are adverse effects noted during other clinical studies.

Cardiovascular: Chest pain (16%), hypertension (11%)

Central nervous system: Pain (32%), chills (21%), absent reflexes (11%), malaise (11%), headache

Dermatologic: Skin rash (21%), pruritus, urticaria

Gastrointestinal: Abdominal pain (47%), gastroenteritis (11%), nausea, vomiting

Hypersensitivity: Angioedema

Neuromuscular & skeletal: Arthralgia (42%)

Ophthalmic: Conjunctivitis (21%), corneal opacity (increased, 11%)

Otic: Otalgia (42%), auditory impairment (11%)

Respiratory: Dyspnea (21%), pharyngitis (11%), nasal congestion (11%), apnea, laryngeal edema, respiratory distress

Miscellaneous: Antibody development (98%), infusion related reaction (56%), umbilical hernia (11%), fever

<1% (Limited to important or life-threatening): Anaphylaxis, bradycardia, bronchospasm, cyanosis, erythema, hypotension, hypoxia, pallor, paresthesia, renal disease (membranous), respiratory failure, shock, spinal cord compression, tachycardia, tachypnea, thrombocytopenia

Commonly reported side effects of galsulfase include: severe infusion related reaction, conjunctivitis, rigors, chest pain, development of igg antibodies, dyspnea, infusion related reaction, otalgia, pain, and pharyngitis. See below for a comprehensive list of adverse effects.

Applies to galsulfase: intravenous solution

General

The most frequently reported adverse reactions included rash, pain, urticaria, pyrexia, pruritus, chills, headache, nausea, vomiting, abdominal pain, and dyspnea.

The most frequently reported adverse reactions requiring interventions are infusion-related reactions.[Ref]

Hypersensitivity

Frequency not reported: Anaphylaxis/anaphylactoid reaction, allergic reaction[Ref]

Dermatologic

Very common (10% or more): Rash (21%), angioedema, urticaria, pruritus
Common (1% to 10%): Erythema[Ref]

Gastrointestinal

Very common (10% or more): Abdominal pain (47%), gastroenteritis (11%), umbilical hernia (11%), nausea, vomiting[Ref]

Other

Very common (10% or more): Infusion reactions (56%), ear pain (42%), pain (32%), chills/rigors (21%), chest pain (16%), malaise (11%), hearing impairment (11%), pyrexia[Ref]

Infusion reactions, which occurred in 56% of patients across 5 clinical studies were defined as adverse reactions occurring during infusions or until the end of the infusion day. Infusion reactions were observed as early as week 1 and as late as week 146 of treatment and occurred during multiple infusions, however, not always in consecutive weeks. The most common signs/symptoms included pruritus, vomiting, abdominal pain, nausea, hypertension, headache, chest pain, erythema, cough, hypotension, angioedema, respiratory distress, tremor, conjunctivitis, malaise, bronchospasm, and arthralgia.[Ref]

Cardiovascular

Very common (10% or more): Hypertension (11%)
Common (1% to 10%): Hypotension
Frequency not reported: Pallor, bradycardia, tachycardia, cyanosis, shock[Ref]

Nervous system

Very common (10% or more): Areflexia (11%), headache
Common (1% to 10%): Tremor
Frequency not reported: Paresthesia[Ref]

Respiratory

Very common (10% or more): Dyspnea (21%), pharyngitis (11%), nasal congestion (11%)
Common (1% to 10%): Apnea, cough, respiratory distress, asthma, bronchospasm
Frequency not reported: Laryngeal edema, hypoxia, tachypnea, sleep apnea
Postmarketing reports: Respiratory failure[Ref]

Musculoskeletal

Very common (10% or more): Arthralgia (42%)[Ref]

Ocular

Very common (10% or more): Conjunctivitis (21%), corneal opacity (11%)[Ref]

Immunologic

Very common (10% or more): Development of antidrug antibodies (98%)[Ref]

During clinical trials, 53 out of 54 patients tested positive for antidrug IgG antibodies within 4 to 8 weeks of treatment. In the placebo controlled study, 19 patients were evaluated for a potential relationship between development of antidrug antibodies to clinical outcome measures. While all 19 patients developed antidrug antibodies, there was no consistent predictive relationship between total antibody titer, neutralizing or IgE antibodies, and infusion-associated reactions, urinary glycosaminoglycan (GAG) levels, or endurance measures.[Ref]

Renal

Postmarketing reports: Membranous nephropathy[Ref]

Hematologic

Postmarketing reports: Thrombocytopenia[Ref]

Some side effects of galsulfase may not be reported. Always consult your doctor or healthcare specialist for medical advice. You may also report side effects to the FDA.

Safety and efficacy have not been established in patients younger than 5 years.

Consult WARNINGS section for additional precautions.