According to a story from EParent, the pharmaceutical company Ultragenyx released a statement confirming that the company’s product MEPSEVII had received approval for public use by the Food and Drug Administration (FDA). This is the first medicine in history certified for the treatment of mucopolysaccharidosis VII in both adults and children.
Mucopolysaccharidosis (MPS) is a metabolic disorder in which lysosomal enzymes are either missing or do not function properly. The enzymes are needed in order to break down certain molecules. There are a total of eleven enzymes that are needed for this process.
The are several different types of MPS that are categorized depending on which enzyme is missing or malfunctioning. In MPS VII, the form for which MEPSEVII was developed, there are varying degress of severity. In rare cases, children may be born with the condition and do not survive beyond a few months. MPS VII is one of the rarest forms of MPS, with only about two hundred known patients. The disease is also known as Sly sydrome. Symptoms include intellectual disability, short stature, skeletal abnormalities, stiffness and limited mobility, and some loss of night and peripheral vision. The condition is caused by a genetic mutation. To learn more about this condition, click here.
The MEPSEVII drug is designed as an enzyme replacement therapy to replace the one that it missing in MPS VII.
The approval of the drug is a pivotal moment for both the medical field and for patients with the disease; prior forms of treatment were limited in their ability to mitigate the condition, and none of them specifically targeted the cause of MPS VII in the way that MEPSEVII will. Due to the fact that it is the first treatment targeted for MPS VII, the treatment was assessed by the FDA with Priority Review, which speeds up to the evaluation process for treatments that are meeting a need that is currently unmet by available therapies.