MFDS approves orphan drug for phenylketonuria treatment under GIFT expedited review

The Ministry of Food and Drug Safety (MFDS) approved the imported orphan drug Sepians powder (sepiapterin) on Sept. 3 for the treatment of hyperphenylalaninemia in patients with phenylketonuria.

The MFDS said it approved the product that day for the treatment of hyperphenylalaninemia in pediatric and adult patients with phenylketonuria.

Phenylketonuria is a rare inherited disorder caused by genetic mutations in the enzyme that breaks down phenylalanine. The resulting accumulation of phenylalanine in the body can cause impaired cognitive function, skin hypopigmentation and developmental disorders.

The drug is converted into tetrahydrobiopterin in the body, where it helps the enzyme that breaks down phenylalanine and lowers blood phenylalanine levels. This improves symptoms of hyperphenylalaninemia.

The MFDS said it expects the drug to provide a new treatment option for an unmet medical need not addressed by existing therapies.

The MFDS designated the drug as No. 49 under the Global Innovative Products on Fast Track (GIFT) support system and conducted an expedited review. GIFT is a program that supports global innovative medical products from the early stages of development so they can be brought to market quickly.

The MFDS said it will continue to rapidly review and approve treatments whose safety and efficacy have been sufficiently confirmed, expanding patients' access to treatment.