Vestronidase alfa, Recombinant human lysosomal beta glucuronidase
Recombinant human lysosomal beta glucuronidase that is a purified enzyme expressed in a CHO cell line. The enzyme is a homotetramer consisted of 4 monomers with 629 amino acids, and holds the same amino acid sequence as human beta-glucuronidase (GUS).
72562.0 Da (Non-glycosylated)
>99% by SDS-Page and HPLC analysis
<0.001 EU per 1 μg of the protein by the LAL method
Indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
Examples of Clinical Use:
Mucopolysaccharidosis VII (MPS VII, Sly syndrome)
In all patients evaluated, the product treatment resulted in reduction of urinary excretion of GAGs including chondroitin sulfate and dermatan sulfate, which was sustained with continued treatment.
Mechanism of action:
Beta-glucuronidase (GUS) is a lysosomal enzyme responsible for degradation of glucuronate-containing glycosaminoglycan (GAG). Resulting lysosomal storage and GAG accumulation in cells from incomplete metabolic degradation of macromolecules leads to damage to multiple tissues and organs. Vestronidase alfa serves as an exogenous source of GUS enzyme for uptake into cellular lysosomes, which is facilitated by the presence of mannose-6-phosphate (M6P) residues on the oligosaccharide chains of the recombinant enzyme. The chains allow binding of the enzyme to cell surface receptors to promote cellular uptake, and targets the lysosomes to achieve catabolism of accumulated GAGs in affected tissues.
Vestronidase alfa, also known as Vestronidase alfa-VJBK or REVESTIVE, is a recombinant enzyme replacement therapy for the treatment of Mucopolysaccharidosis VII (MPS VII, also known as Sly syndrome). MPS VII is a rare inherited metabolic disorder mainly caused by a defect in the enzyme N-acetylglucosamine-6-sulfatase (GNS), which is responsible for the breakdown of glucosamine sulfate. In patients with MPS VII, the loss of this enzyme leads to the accumulation of this waste product in cells and tissues. Symptoms of MPS VII include organ damage, intellectual disability, skeletal malformations, and respiratory problems. Vestronidase alfa is an enzyme-replacement therapy that is administered intravenously and is made from a derivative of the human enzyme glycosylase. It can replace the missing GNS enzyme and help to break down glucosamine sulfate waste and reduce its accumulation in the body. This enzyme replacement therapy aims to slow disease progression and improve the quality of life in patients with MPS VII. Vestronidase alfa, usually as part of a long-term treatment, enzyme replacement therapy is needed on a regular basis. Use of the drug safety and efficacy has been demonstrated in clinical trials and has obtained related regulatory approval. It is important that patients treated with Vestronidase alfa do so under the supervision of a physician and with regular follow-up as recommended by the physician.
2. Clinical use and mechanism
The clinical use of Vestronidase alfa is for the treatment of Mucopolysaccharidosis VII (MPS VII, also known as Sly syndrome), a rare inherited metabolic disorder. This drug can improve the quality of life of patients with MPS VII and slow their disease progression. Mechanistically, Vestronidase alfa is an enzyme replacement therapy that is a recombinant human glycosylase derivative. In MPS VII patients, lack of the N-acetylglucosamine-6-sulfatase (GNS) enzyme leads to the accumulation of glucosamine sulfate waste products in the body, which affects many different cells and tissues. Vestronidase alfa could replace the missing GNS enzyme and play a similar role at the site of its deletion, decomposing glucosamine sulfate waste and reducing its accumulation in the body, thereby slowing the disease progression of MPS VII. In clinical trials, the use of this drug has improved the performance of MPS VII patients in terms of exercise capacity, mental health, fatigue level, and respiratory function.
3. Advantages and Disadvantages
Vestronidase alfa is an enzyme replacement therapy used to treat MPS VII. Here are some advantages and disadvantages of Vestronidase alfa in clinical use: Advantages: (1) Disease management: Vestronidase alfa can reduce the accumulation of glucosamine sulfate waste in MPS VII patients, which helps to slow the progression of the disease and improve the quality of life of patients. (2) Safety: The drug has been shown to be safe and effective through clinical trials and regulatory approval. (3) Evidence support: The efficacy and safety of Vestronidase alfa have been confirmed in clinical trials, and relevant data support its clinical application. Disadvantages: (1) Frequent treatment: Vestronidase alfa usually requires intravenous administration and may require frequent treatment and regular enzyme-replacement therapy, which may impose an additional burden on the patient and family. (2) Drug costs: Enzyme replacement therapy is usually more expensive, and may have on the financial condition of patients and the health care system. Although Vestronidase alfa has several advantages in the treatment of MPS VII, the burden on patients and the healthcare system also needs to be considered. Therefore, before using the drug, the efficacy, safety, and should be considered factors such as economic costs, together with the doctor for individual decision-making and treatment plan.
4. Current research situation
Currently, Vestronidase alfa in other diseases associated with MPS VII efficacy and safety research is underway, the study aims to enlarge the indications of a drug, to cover a wider range of patients. Several clinical trials have been conducted to evaluate the efficacy and pharmacokinetics of Vestronidase alfa in children and adults, with the main goal of better understanding the efficacy, dosage, and safety of the drug in order to provide more effective treatment options for patients. Efforts are also underway to find more optimized Vestronidase alfa regimens to improve efficacy and reduce side effects, which may involve adjustments in drug dosage, frequency of administration, and duration of treatment.
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