For the treatment of a rare, genetic disease, known as mucopolysaccharidosis type VII or Sly syndrome, a drug, named Mepsevii has been recently approved by the U.S. Food and Drug Administration (FDA).
Worldwide, this disease has affected approximately 150 people. Sly syndrome affects the skeletal system of a person, which worsens with age, and also leads to abnormalities like short stature. The symptoms and life expectancy of the patient vary widely. This disease is also found to be linked with other health conditions, such as heart valve defects, liver enlargement, narrowing of the respiratory tract.
Mepsevii acts by reversing a condition of enzyme deficiency, which results in the accumulation of toxic substances in the body.
This was tested through a clinical trial, which included 23 people, aged 5 months to 25 years. Dr. Julian Beitz, one of the directors of FDA’s Drug Evaluation Department, said that before the approval of the drug, people generally inherited the symptoms and conditions that had no cure until then.
There are several side effects of the drug that includes an allergic reaction at the site of infusion, diarrhea, rashes, and sometimes severe anaphylaxis reaction.