ELAPRASE® (idursulfase) is a prescription medicine for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II), which was approved by the Food and Drug Administration (FDA) in 2006.
The active substance in ELAPRASE is an enzyme that breaks down glycosaminoglycans (GAG). Due to an enzyme deficiency, these GAGs accumulate inside people with Hunter syndrome, causing the signs and symptoms of the condition. For example, the build-up of GAGs within organs such as the liver and spleen results in enlarged organ size.
ELAPRASE is an "enzyme replacement therapy" (ERT) because it is a formulation of the enzyme called iduronate-2-sulfatase (I2S) which is missing or deficient in people with Hunter syndrome.
ELAPRASE is a purified form of the I2S enzyme produced by recombinant DNA technology in a human cell line.
ELAPRASE is the first and only FDA-approved ERT for Hunter syndrome.
ELAPRASE is a global brand with
over 15 years of real-world experience
ELAPRASE is available in 77 countries* and has been FDA approved since 2006
As of 2020, ELAPRASE was approved in 77* other countries after receiving respective marketing authorizations, and so is available to patients around the world.
ELAPRASE is the first and only ERT with over 15 years' clinical experience, approved for the treatment of Hunter syndrome in the United States.
As of 2020, ELAPRASE was approved in 77* other countries after receiving respective marketing authorizations, and so is available to patients around the world.
ELAPRASE hits 10-year mark. As of July 2016, 1,200 patients, from 134 clinics, in 33 countries had been enrolled in the Hunter Outcome Survey (HOS), making it the largest global source of data on Hunter syndrome.
10-year anniversary of HOS data and patient enrollment.
U.S. Prescribing Information updated to include information about the use of ELAPRASE in children aged 16 months to 5 years of age.
In patients 16 months to 5 years old, ELAPRASE did not show improvement in disease-related symptoms or long term clinical result; however, treatment with ELAPRASE has reduced spleen size similarly to patients 5 years and older.
It is not known if ELAPRASE is safe and effective in children under 16 months old.
HOS publication: Evaluated data of idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the HOS.
ELAPRASE receives FDA approval for use in patients with MPS II aged 5 years or older, based on data from the pivotal clinical trial in MPS II patients aged 5–31 years.
HOS began patient enrollment. HOS is a Takeda-sponsored, global, long-term observational survey of MPS II patients established in order to better understand the variability of symptoms and progression of MPS II in the population.
Idursulfase enzyme replacement therapy clinical trial program initiated, beginning with a Phase I/II trial.
Development of idursulfase, a purified form of the human I2S enzyme produced by recombinant DNA technology in a continuous human cell line.
I2S enzyme amino acid sequence deduced from the gene sequence. Discovery of enzyme replacement therapy as a possible treatment for Hunter syndrome.
Hunter corrective factor found (later known to be I2S, the enzyme deficient or malfunctioning in MPS II patients).
First cases of Hunter syndrome characterized (later also known as MPS II, a member of the MPS family of inherited disorders of GAG catabolism).
*indication and risk information may vary by country.
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RISK OF SERIOUS ALLERGIC REACTIONS
Some patients have experienced serious allergic reactions (including life-threatening anaphylactic reactions) during and up to 24 hours after treatment, regardless of how long they were taking ELAPRASE. Anaphylactic reactions are immediate and include breathing problems, low oxygen levels, low blood pressure, hives and/or swelling of the throat or tongue. If a patient (you or your child) has experienced an anaphylactic reaction, the patient may require an extended period of observation by the patient’s healthcare team. If you or your child has breathing problems, a fever, or a respiratory illness, you or your child may be at risk of life-threatening worsening of those conditions due to allergic reactions from ELAPRASE. Your healthcare team should be advised of those conditions before treatment with ELAPRASE because the information may affect the timing of ELAPRASE treatment.
You or your child should be closely watched during and after ELAPRASE treatment and you should confirm with your healthcare team in advance of treatment that it is prepared to manage serious allergic reactions, including anaphylactic reactions. Tell your healthcare team immediately if any signs of an allergic reaction happen. Those signs may include breathing problems, low blood pressure, rash, hives, itching, flushing, fever and/or headache.
When serious allergic reactions happened during clinical trials, later ELAPRASE treatments were managed with allergy-controlling drugs before or during treatment, a slower rate of ELAPRASE treatment, and/or early discontinuation of treatment.
Children with serious genetic mutations may be at risk for allergic reactions, serious side effects and antibody development. In a clinical study of children 7 years and younger, patients with certain types of genetic mutations experienced a higher number of allergic reactions, serious side effects, and development of an immune response to treatment. This immune response may interfere with the effectiveness of ELAPRASE. Talk to your healthcare team about whether you or your child may be at risk.
If you or your child has breathing problems, other respiratory illness, heart problems, or susceptibility to fluid overload, you or your child may be at higher risk of fluid overload during ELAPRASE treatment. Your healthcare team should be advised of those problems before treatment and you should confirm with your healthcare team in advance of treatment that it is appropriately trained to watch for signs of fluid overload and provide the necessary medical support. Patients at risk for fluid overload may require longer observation time.
What are possible side effects of ELAPRASE?
The most common side effects of ELAPRASE include:
-
In patients aged 5 and older:
- - Headache
- - Itching
- - Muscle and bone pain
- - Hives
- - Diarrhea
- - Cough
-
In patients aged 7 years or younger:
- - Fever
- - Rash
- - Vomiting
- - Hives
The most common side effects needing medical attention were allergic reactions, and included rash, hives, itching, flushing, fever, and headache. Tell your healthcare team immediately if any signs of an allergic reaction happen. These are not all the possible side effects of ELAPRASE.
What is ELAPRASE® (Idursulfase)?
ELAPRASE is a prescription medicine for patients with Hunter syndrome. ELAPRASE has been shown to improve walking ability in patients 5 yrs and older. In patients 16 months to 5 yrs old, ELAPRASE did not show improvement in disease-related symptoms or long term clinical result; however, treatment with ELAPRASE has reduced spleen size similarly to patients 5 yrs and older. It is not known if ELAPRASE is safe and effective in children under 16 months old.
RISK OF SERIOUS ALLERGIC REACTIONS
Some patients have experienced serious allergic reactions (including life-threatening anaphylactic reactions) during and up to 24 hours after treatment, regardless of how long they were taking ELAPRASE. Anaphylactic reactions are immediate and include breathing problems, low oxygen levels, low blood pressure, hives and/or swelling of the throat or tongue. If a patient (you or your child) has experienced an anaphylactic reaction, the patient may require an extended period of observation by the patient’s healthcare team. If you or your child has breathing problems, a fever, or a respiratory illness, you or your child may be at risk of life-threatening worsening of those conditions due to allergic reactions from ELAPRASE. Your healthcare team should be advised of those conditions before treatment with ELAPRASE because the information may affect the timing of ELAPRASE treatment.
You or your child should be closely watched during and after ELAPRASE treatment and you should confirm with your healthcare team in advance of treatment that it is prepared to manage serious allergic reactions, including anaphylactic reactions. Tell your healthcare team immediately if any signs of an allergic reaction happen. Those signs may include breathing problems, low blood pressure, rash, hives, itching, flushing, fever and/or headache.
When serious allergic reactions happened during clinical trials, later ELAPRASE treatments were managed with allergy-controlling drugs before or during treatment, a slower rate of ELAPRASE treatment, and/or early discontinuation of treatment.
Children with serious genetic mutations may be at risk for allergic reactions, serious side effects and antibody development. In a clinical study of children 7 years and younger, patients with certain types of genetic mutations experienced a higher number of allergic reactions, serious side effects, and development of an immune response to treatment. This immune response may interfere with the effectiveness of ELAPRASE. Talk to your healthcare team about whether you or your child may be at risk.
If you or your child has breathing problems, other respiratory illness, heart problems, or susceptibility to fluid overload, you or your child may be at higher risk of fluid overload during ELAPRASE treatment. Your healthcare team should be advised of those problems before treatment and you should confirm with your healthcare team in advance of treatment that it is appropriately trained to watch for signs of fluid overload and provide the necessary medical support. Patients at risk for fluid overload may require longer observation time.
What are possible side effects of ELAPRASE?
The most common side effects of ELAPRASE include:
-
In patients aged 5 and older:
- - Headache
- - Itching
- - Muscle and bone pain
- - Hives
- - Diarrhea
- - Cough
-
In patients aged 7 years or younger:
- - Fever
- - Rash
- - Vomiting
- - Hives
The most common side effects needing medical attention were allergic reactions, and included rash, hives, itching, flushing, fever, and headache. Tell your healthcare team immediately if any signs of an allergic reaction happen. These are not all the possible side effects of ELAPRASE.
What is ELAPRASE® (Idursulfase)?
ELAPRASE is a prescription medicine for patients with Hunter syndrome. ELAPRASE has been shown to improve walking ability in patients 5 yrs and older. In patients 16 months to 5 yrs old, ELAPRASE did not show improvement in disease-related symptoms or long term clinical result; however, treatment with ELAPRASE has reduced spleen size similarly to patients 5 yrs and older. It is not known if ELAPRASE is safe and effective in children under 16 months old.
