The Ministery of Food and Drug Safety said on the 12th that it approved a therapy used to treat non-central nervous system symptoms in patients with the rare disease "alpha-mannosidosis," which causes facial and skeletal abnormalities and immunodeficiency due to a deficiency of a specific enzyme.
Alpha-mannosidosis is a disease that occurs when oligoses containing mannose are not properly broken down and accumulate in the body due to a deficiency of alpha-mannosidase within lysosomes. This leads to various systemic symptoms, including facial and skeletal abnormalities and decreased immune function.
The therapy approved this time is "Ramzede (ingredient name velmanase alfa)," developed by the Italian rare disease specialist Chiesi, and it is currently the only approved medicine for alpha-mannosidosis. Kwangdong Pharmaceutical introduced it in Korea. The approved dose is 10 mg.
Ramzede is a recombinant alpha-mannosidase preparation that supplements the deficient enzyme in patients to reduce the accumulation of mannose-containing oligosaccharides in organs and alleviate non-central nervous system symptoms.
Until now, there had been no approved therapy in Korea for patients with alpha-mannosidosis, but this approval is expected to provide treatment options for these patients.
The Ministery of Food and Drug Safety said, "We will continue to work to ensure that new therapies can be supplied quickly to rare disease patients based on our regulatory science expertise."