US FDA approved new drug Mepsevii that contains active pharmaceutical ingredient vestronidase alfa-vjbk for the treatment of metabolic disorder called as mucopolysaccharidosis type VII (MPS VII) which also described as Sly syndrome. This drug can be used in both adult as well as pediatric patients. Though, mucopolysaccharidosis type VII is rare kind of genetic disorder but it is progressively affects body tissues and organs.
Mucopolysaccharidosis Type VII or Sly syndrome is genetically induced disease characterized as deficiency of enzyme β-glucuronidase. Due to deficiency of this enzyme deposition of complex carbohydrates e.g mucopolysaccharides occur inside the tissues and organs of the body that's why it is termed as Mucopolysaccharidosis Type VII. It is basically an autosomal recessive lysosomal storage disease. It also named after an American biochemist William S. Sly who first described this disease (Sly syndrome).
Most commonly observed symptoms associated with Mucopolysaccharidosis Type VII or MPS include macrocephaly, corneal opacity, anteverted nostrils, cleft palate, kyphosis or hook like deformity in thoracic area. In severe cases pulmonary infection, spleen enlargement and liver enlargement may occur. In newborn, this may cause growth retardation and affect motor functions of body.
Mepsevii is an enzyme replacement therapy that works by replacing the missing enzyme due to which accumulation of complex carbohydrates reduce to a great extent and patient also gets relief in severity of symptoms.
Mepsevii/vestronidase alfa-vjbk Side effects
Following side effects were observed during clinical trail of this drug
- Rashes on skin
- Reaction at injection site
- Skin irritation
Mepsevii/vestronidase alfa-vjbk Uses/Prescribing Information
US FDA approved this drug for the treatment of rare genetic disorder mucopolysaccharidosis type VII (MPS VII) for both adults and child. This drug is an enzyme replacement therapy that works by replacing the missing enzyme.
Mepsevii/vestronidase alfa-vjbk Dosage
This drug is formulated in parenteral dosage form with potency of 2mg/mL (10mg/5mL single-dose vial). According to health authorities it is recommended that administer injection of any non-sedating antihistamine one hour before injecting Mepsevii to reduce the risk of reaction or hypersensitivity.