VPRIV (velaglucerase alfa) 400 Units powder for solution for infusion

VPRIV (velaglucerase alfa) is an enzyme replacement therapy (ERT) used to treat Gaucher disease, a rare genetic disorder. Gaucher disease results from a deficiency in the enzyme glucocerebrosidase, leading to the accumulation of fatty substances in certain organs, especially the spleen and liver. VPRIV is specifically designed to replace the deficient enzyme, thereby reducing symptoms and preventing complications associated with the disease.

How It Works

VPRIV contains velaglucerase alfa, a recombinant form of the human enzyme glucocerebrosidase. This enzyme plays a critical role in breaking down a specific fatty substance called glucocerebroside into glucose and ceramide. In patients with Gaucher disease, the deficiency of glucocerebrosidase leads to the buildup of glucocerebroside in the lysosomes of macrophages, resulting in the formation of Gaucher cells. These cells accumulate in various organs, causing the clinical manifestations of Gaucher disease. Here’s how VPRIV works:

1. Replaces Deficient Enzyme: Velaglucerase alfa provides a functional version of the enzyme that Gaucher patients lack, helping to break down glucocerebroside.
2. Reduces Gaucher Cells: By breaking down excess glucocerebroside, VPRIV reduces the number of Gaucher cells, alleviating symptoms and preventing damage to organs such as the spleen, liver, and bones.
3. Alleviates Symptoms: The treatment helps reduce symptoms such as anemia, fatigue, bone pain, and organ enlargement, improving the patient’s quality of life.
4. Infusion Process: VPRIV is administered via intravenous infusion, allowing the enzyme to circulate through the bloodstream and reach affected organs.

Clinical Efficacy

Clinical trials have shown that VPRIV effectively reduces spleen and liver size, improves blood counts, and alleviates bone pain in patients with Gaucher disease. The therapy offers a well-tolerated and effective option for managing this chronic condition, with improvements often observed within the first six months of treatment.

Side Effects

While VPRIV is generally well-tolerated, some patients may experience side effects ranging from mild to severe. It’s essential to monitor for these side effects and report any concerns to a healthcare provider.

Common Side Effects

• Infusion-related reactions: These may occur during or after the infusion and include:
  • Headache
  • Dizziness
  • Fever
  • Chills
  • Fatigue
  • Nausea
  • Back pain
  • Joint pain
• Respiratory symptoms: Upper respiratory tract infections, nasal congestion, and cough.
• Gastrointestinal symptoms: Abdominal pain, diarrhea, and vomiting.
• Skin reactions: Rash, itching, and redness at the injection site.

Serious Side Effects

• Hypersensitivity reactions: Some patients may experience severe allergic reactions, including anaphylaxis, which require immediate medical attention.
• Elevated liver enzymes: Indicating potential liver inflammation or damage.
• Low blood pressure: During or after the infusion.

Patients experiencing severe side effects should contact their healthcare provider promptly to address these issues and adjust the treatment plan as needed.

Indications

VPRIV is specifically indicated for long-term enzyme replacement therapy in patients with type 1 Gaucher disease.

Approved Indications

1. Type 1 Gaucher Disease: VPRIV is used to treat non-neuronopathic Gaucher disease, characterized by:
   • Enlargement of the spleen and liver
   • Anemia
   • Thrombocytopenia (low platelet count)
   • Bone disease, including pain and fractures

By replacing the deficient enzyme, VPRIV helps manage these symptoms and prevent disease progression.

Contraindications

While VPRIV is effective for treating Gaucher disease, certain contraindications should be considered to ensure patient safety.

Contraindications

1. Hypersensitivity: Patients with known hypersensitivity to velaglucerase alfa or any excipients in VPRIV should not receive this medication. Hypersensitivity reactions can include anaphylaxis and other severe allergic reactions.
2. Severe Cardiac or Pulmonary Conditions: Patients with severe cardiac or pulmonary conditions should be monitored closely, as infusion reactions can exacerbate these conditions.
3. Pregnancy and Breastfeeding: The effects of VPRIV during pregnancy and breastfeeding are not well established. Therefore, it should be used with caution in pregnant or breastfeeding women, only if the potential benefit justifies the potential risk to the fetus or infant.
4. Immune-mediated Reactions: Patients with a history of immune-mediated reactions to other enzyme replacement therapies should be monitored closely during VPRIV treatment.

   Price in Different Countries

Country	Price (USD) Per 400 Units	Reference Link
United States	$18,000 – $20,000	GoodRx
Canada	$15,000 – $17,000	CanadaDrugs
United Kingdom	£14,000 – £16,500	NHS
Australia	$16,000 – $18,500	PBS
Germany	€15,500 – €18,000	Apotheken Umschau

Top 5 Global Brands

VPRIV is part of a growing field of enzyme replacement therapies, with several global pharmaceutical companies developing similar treatments. Here are the top 5 global brands known for their contributions to enzyme replacement therapy:

1. Takeda Pharmaceutical Company: The developer of VPRIV and a leader in biotechnology and pharmaceutical innovation, particularly in rare diseases and enzyme replacement therapies.
2. Sanofi Genzyme: A pioneer in rare diseases and enzyme replacement therapy, offering treatments for conditions like Gaucher disease and Fabry disease.
3. BioMarin Pharmaceutical: Known for its focus on genetic and rare diseases, with a strong emphasis on enzyme replacement therapies for various conditions.
4. Shire (now part of Takeda): Previously a significant player in enzyme replacement therapy, with a portfolio that included treatments for Gaucher and other lysosomal storage disorders.
5. Amicus Therapeutics: Specializes in developing therapies for rare metabolic diseases, including innovative approaches to enzyme replacement therapy.