Elaprase (idurskfase) 6mg/3ml injection

Elaprase is a prescription medication used as enzyme replacement therapy for patients with Hunter syndrome (also known as mucopolysaccharidosis II or MPS II). Hunter syndrome is a rare, genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase, which is crucial for the breakdown of glycosaminoglycans (GAGs) in the body. Without this enzyme, GAGs accumulate in cells and tissues, leading to a range of serious health issues, including organ dysfunction, physical deformities, and developmental delays. Elaprase is manufactured by Shire Human Genetic Therapies, a part of Takeda Pharmaceutical Company, and is designed to replace the missing enzyme, helping to reduce the buildup of GAGs and alleviate the symptoms of Hunter syndrome.

How It Works

Elaprase works by providing a synthetic form of the enzyme iduronate-2-sulfatase, which is deficient or missing in individuals with Hunter syndrome. Here’s a detailed explanation of its mechanism of action: 1. **Enzyme Replacement**: – **Idursulfase**: Elaprase contains idursulfase, a recombinant form of the naturally occurring human enzyme iduronate-2-sulfatase. This enzyme is produced using recombinant DNA technology in a continuous human cell line, ensuring that the recombinant enzyme closely resembles the naturally occurring enzyme in structure and function. 2. **Breakdown of Glycosaminoglycans (GAGs)**: – The primary function of iduronate-2-sulfatase is to catalyze the hydrolysis of sulfate groups from GAGs such as dermatan sulfate and heparan sulfate. These molecules are typically broken down and recycled in the lysosomes, the cell’s recycling centers. – In patients with Hunter syndrome, the deficiency of iduronate-2-sulfatase leads to the accumulation of these GAGs, causing cellular and tissue damage. By introducing idursulfase, Elaprase helps to break down these accumulated GAGs, reducing their storage in lysosomes. 3. **Reduction of Symptoms**: – By reducing the storage of GAGs in tissues and organs, Elaprase helps alleviate many symptoms associated with Hunter syndrome. This includes reducing organomegaly (enlargement of organs such as the liver and spleen), improving joint flexibility, and enhancing overall growth and physical health. – Elaprase may also help slow the progression of symptoms, potentially improving quality of life and extending life expectancy for patients with Hunter syndrome.

Indications

Elaprase is indicated for:

• Hunter Syndrome (Mucopolysaccharidosis II or MPS II):
  • **Enzyme Replacement Therapy**: Elaprase is used for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Hunter syndrome. It helps manage and reduce the symptoms associated with this genetic disorder by breaking down accumulated GAGs in the body.

Side Effects

Elaprase may cause a range of side effects, from mild to severe. Common and serious side effects include:

• Common Side Effects:
  • Headache
  • Fever
  • Abdominal pain
  • Nausea
  • Diarrhea
  • Vomiting
  • Joint pain
  • Rash
  • Cough
  • Itching
  • Flushing
  • Ear infections
  • Upper respiratory tract infection
• Serious Side Effects:
  • **Infusion-Related Reactions**: Some patients may experience infusion-related reactions, which can be mild to severe. Symptoms may include flushing, chest pain, shortness of breath, fever, or swelling. Pre-medication with antihistamines or corticosteroids may be necessary to minimize these reactions.
  • **Anaphylaxis**: A severe allergic reaction can occur in some patients. Symptoms may include difficulty breathing, swelling of the face or throat, and a drop in blood pressure. Immediate medical attention is required if anaphylaxis occurs.
  • **Antibody Development**: Some patients may develop antibodies against idursulfase, potentially reducing the drug’s effectiveness. Monitoring for the development of these antibodies is recommended.
  • **Severe Hypersensitivity Reactions**: Symptoms may include hives, difficulty breathing, and swelling of the face, lips, or tongue. Immediate medical attention is necessary.
  • **Thromboembolic Events**: Although rare, there is a risk of blood clots forming in veins or arteries, which can lead to serious conditions like deep vein thrombosis (DVT) or pulmonary embolism (PE).

Contraindications

Elaprase is contraindicated in the following situations:

• Hypersensitivity: Patients with known hypersensitivity to idursulfase or any components of the product should not use Elaprase.
• Severe Allergic Reactions: Individuals who have experienced severe or life-threatening hypersensitivity reactions to Elaprase should avoid further use unless an appropriate management protocol is in place.

Pricing Information

Below is a table detailing the price of Elaprase in various countries:

Country	Price (per vial)	Reference
United States	$6,000 – $8,000	GoodRx
Canada	CAD $7,500 – $9,000	DrugBank
United Kingdom	£5,500 – £7,000	NHS
Australia	AUD $8,000 – $10,000	PBS
Germany	€5,800 – €7,500	Apotheken Umschau

Top 5 Global Brands

In addition to Elaprase, several pharmaceutical companies produce enzyme replacement therapies for various lysosomal storage disorders. Here are some of the top global brands:

• Aldurazyme (laronidase) – Sanofi Genzyme
• Fabrazyme (agalsidase beta) – Sanofi Genzyme
• Cerezyme (imiglucerase) – Sanofi Genzyme
• Vpriv (velaglucerase alfa) – Takeda
• Replagal (agalsidase alfa) – Shire/Takeda