MFDS Approves Rare Disease Treatment Sepiansan

[Financial News] The Ministry of Food and Drug Safety approved Sepiansan (sepiapterin), an orphan drug used to treat hyperphenylalaninemia in pediatric and adult patients with phenylketonuria, on the 3rd.
Phenylketonuria is a rare inherited disorder in which phenylalanine accumulates in the body due to genetic mutations in phenylalanine hydroxylase (PAH), the enzyme that breaks down phenylalanine. If left untreated, it can cause cognitive impairment and developmental disabilities.
Sepiansan is converted into tetrahydrobiopterin in the body, where it helps PAH function. Tetrahydrobiopterin is a cofactor required by enzymes involved in breaking down amino acids. By supporting this process, the drug promotes the conversion of phenylalanine into tyrosine and can lower blood phenylalanine levels.
The Ministry of Food and Drug Safety designated Sepiansan as the 49th product under the Global Innovative Products Fast Track Review Support System (GIFT) and conducted an expedited review. GIFT is a program that provides regulatory support from the early stages of development so that globally innovative medical products can be approved quickly.
The Ministry of Food and Drug Safety expects the approval to provide patients with phenylketonuria with a new treatment option. It also plans to expedite reviews of treatments whose safety and efficacy have been confirmed, based on regulatory science, to expand patients’ access to care.
[email protected] Jung Sang-hee Reporter