Idursulfase

Idursulfase is used to treat some of the symptoms of a genetic condition called Hunter's syndrome, also called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis).

Hunter syndrome 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 loss, and changes in mental or physical abilities.

Idursulfase may improve walking ability in people with this condition. However, this medication is not a cure for Hunter syndrome.

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

Some side effects may occur during the idursulfase infusion, or up to 24 hours afterward. Get emergency medical help if you have any of these signs of an allergic reaction: feeling like you might pass out; hives; trouble breathing; seizure (convulsions); swelling of your face, lips, tongue, or throat.

You may be more likely to have a reaction to idursulfase if you have a breathing disorder.

You should not receive this medication if you are allergic to idursulfase.

To make sure idursulfase is safe for you, tell your doctor if you have asthma or any other lung problems. You may be more likely to have a reaction to idursulfase if you have a breathing disorder.

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

FDA pregnancy category C. It is not known whether idursulfase will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medication.

It is not known whether idursulfase passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

Some side effects may occur during the idursulfase infusion, or up to 24 hours afterward. Get emergency medical help if you have any of these signs of an allergic reaction: feeling like you might pass out; hives; trouble breathing, seizure (convulsions); swelling of your face, lips, tongue, or throat.

It may still be possible for you to receive idursulfase even after you have had a reaction to it. There are other medications that can be given to you before your idursulfase infusion to help prevent symptoms of a reaction.

Common side effects may include:

• vomiting, diarrhea;
• headache;
• fever, cough;
• itching, mild skin rash; or
• flushing (warmth, redness, or tingly feeling).

This is not a complete list of side effects and others may occur. Tell your doctor about any unusual or bothersome side effect. You may report side effects to FDA at 1-800-FDA-1088.

>10%

Pyrexia (63%)

Headache (59%)

Arthralgia (31%)

Limb pain (28%)

Pruritus (28%)

Hypertension (25%)

Malaise (22%)

Visual disturbance (22%)

Wheezing (19%)

Abscess (16%)

Chest wall musculoskeletal pain (16%)

Musculoskeletal dysfunction (16%)

Urticaria (16%)

AE resulting from injury (13%)

Anxiety (13%)

Iirritability (13%)

Atrial abnormality (13%)

Dyspepsia (13%)

Infusion site edema (13%)

Pruritic rash (13%)

Skin disorder (13%)

Superficial injury (13%)

Frequency Not Defined

Angioedema

Loss of conaciousness

Seizure

Cardiac arrhythmia

Cyanosis

Black Box Warnings

Anaphylactoid reactions, which may be life threatening, have been observed in some patients during idursulfase infusions

Anaphylaxis, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have been reported to occur during and after infusions, regardless of duration of the course of treatment

Appropriate medical support should be readily available when idursulfase is administered

Biphasic anaphylactic reactions have also been observed after idursulfase administration, and patients who have experienced anaphylactic reactions may require prolonged observation

Patients with compromised respiratory function or acute respiratory distress may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring

Contraindications

None listed in the manufacturer's label

Cautions

Risk of anaphylactoid reactions; monitor patients with compromised respiratory function/acute respiratory disease

Patients with compromised respiratory function or acute febrile or respiratory illness at the time of infusion may be at higher risk of life-threatening complications from hypersensitivity reactions

Risk of hypersensitivity and antibody development is higher in individuals with seere genetic mutations (ie, complete gene deletion, large gene rearrangement, nonsense, frameshift or splice site mutations)

Caution when administering to patients susceptible to fluid overload, or patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function for whom fluid restriction is indicated; risk of serious exacerbation of cardiac or respiratory status during infusions

Development of anti-idursulfase IgG antibodies reported in 51% of patients, which may increase incidence of infusion-related reactions

A registry has been created to track adverse effects, and monitor therapeutic responses during long-term treatment; may contact 1-866-888-0660 or www.eleprase.com

Take idursulfase exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The Idursulfase dose your doctor recommends will be based on the following (use any or all that apply):

• the condition being treated
• other medical conditions you have
• other medications you are taking
• how you respond to this medication
• your weight
• your height
• your age
• your gender

Idursulfase is available in the following doses:

• Idursulfase 2 Mg/ml Intravenous Solution

Idursulfase is used to treat some of the symptoms of a genetic condition called Hunter's syndrome, also called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis).

Hunter syndrome 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 loss, and changes in mental or physical abilities.

Idursulfase may improve walking ability in people with this condition. However, this medication is not a cure for Hunter syndrome.

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

Usual Adult Dose for Mucopolysaccharidosis Type II:

0.5 mg/kg IV once a week

Comment:
-Clinical studies did not include patients older than 31 years and it is therefore unknown if they respond differently than younger patients.

Use: For the treatment of Hunter syndrome (Mucopolysaccharidosis II [MPS II])

Usual Pediatric Dose for Mucopolysaccharidosis Type II:

16 months or older:
0.5 mg/kg IV once a week

Use: For the treatment of Hunter syndrome (MPS II)

No dosage adjustment provided in manufacturer’s labeling.

Allow vials to reach room temperature prior to preparation. Dilute dose in NS 100 mL. Mix gently, do not shake.

Concerns related to adverse effects:

• Hypersensitivity/anaphylactoid reactions: [U.S. Boxed Warning]: Serious hypersensitivity reactions, including fatal life-threatening anaphylactic reactions, have been reported during and within 24 hours after infusion. Anaphylaxis may present as respiratory distress, hypoxia, hypotension, urticaria, and/or tongue/throat angioedema. Monitor closely during and after infusion. Appropriate medical support should be readily available. Patients with compromised respiratory function or acute respiratory disease are at risk of respiratory disease exacerbation due to hypersensitivity; additional monitoring may be required. Discontinue immediately if anaphylactic or acute reaction occurs. Patients experiencing initial severe or refractory reactions may need prolonged monitoring. Antihistamines, corticosteroids and/or decreased infusion rates may be used to manage subsequent infusions.

• Antibody formation: Development of anti-idursulfase IgG antibodies has been reported in 51% of patients; may increase incidence of hypersensitivity reactions.

Disease-related concerns:

• Acute febrile/respiratory illness: Hypersensitivity reactions may occur. Use caution and consider delaying treatment in patients with compromised respiratory function or acute febrile or respiratory illness; may be at increased risk for life-threatening complications from hypersensitivity reactions.

• Fluid overload: Use with caution in patients at risk for fluid overload or in conditions where fluid restriction is indicated (eg, acute underlying respiratory illness, compromised cardiac and/or respiratory function); conditions may be exacerbated during infusion. Extended observation may be necessary for some patients.

• Genetic mutations: Use with caution in patients with severe genetic mutations (eg, complete gene deletion, large gene rearrangement, nonsense, frameshift or splice site mutations); may increase risk of hypersensitivity reactions, serious adverse reactions, and antibody development.

Other warnings/precautions:

• Registry: Patients and healthcare providers are encouraged to participate in the Hunter Outcome Survey, intended to monitor disease progression, patient outcomes, and long-term effects of therapy. For more information, refer to www.elaprase.com or call OnePathsm at 1-866-888-0660.

Commonly reported side effects of idursulfase include: abscess, chest wall pain, dyspepsia, infusion related reaction, pruritic rash, pruritus, urticaria, and visual disturbance. See below for a comprehensive list of adverse effects.

Applies to idursulfase: intravenous solution

Along with its needed effects, idursulfase may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur while taking idursulfase:

• Bone or muscle pain
• chest pain
• chills
• cough
• fast, pounding, or irregular heartbeat or pulse
• feeling of warmth
• fever
• headache
• hives or welts
• itching
• rash
• redness of the face, neck, arms, and occasionally, upper chest
• redness of the skin
• sneezing
• sore throat
• tightness in the chest
• unusual tiredness or weakness

• Blurred vision
• confusion
• dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
• sweating

• Difficulty with swallowing
• dilated neck veins
• extreme fatigue
• heart stops
• irregular breathing
• no breathing
• no pulse or blood pressure
• puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
• unconscious
• weight gain

Some side effects of idursulfase may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

• Change in hearing
• diarrhea
• ear drainage
• earache or pain in the ear

• Nausea
• vomiting