MFDS approves rare disease drug Sepiancer Powder for phenylketonuria, expanding treatment options

The Ministry of Food and Drug Safety (MFDS) approved the imported rare disease drug Sepiancer Powder (sepiapterin) on the 3rd for the treatment of hyperphenylalaninemia in patients with phenylketonuria.

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

Phenylketonuria is a rare inherited disorder in which a genetic mutation in the enzyme that breaks down phenylalanine, a type of amino acid, causes phenylalanine to accumulate in the body, potentially leading to impaired cognitive function and developmental disabilities.

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

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

The MFDS designated the drug as GIFT No. 49 under the Global Innovative Products on Fast Track (GIFT) support system and conducted an expedited review to enable its rapid introduction into clinical practice.

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.