Kwangdong Pharmaceutical Secures Domestic Approval for Fabry Disease Treatment

Kwangdong Pharmaceutical is accelerating its expansion into the rare disease treatment market following the domestic approval of its Fabry disease treatment. As the supply of high-cost rare drugs stabilizes, competition for the introduction of rare disease treatments in South Korea is expected to intensify.

On May 22, Kwangdong Pharmaceutical announced that it received product approval from the Ministry of Food and Drug Safety for its Fabry disease treatment, Elprazole (active ingredient: pegunigalsidase alfa). This medication is used to treat Fabry disease, a rare genetic lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. Symptoms can include angiokeratomas, abdominal pain, proteinuria, peripheral pain, abnormal sweating, and hearing and vision impairments.

This approval marks a significant step for Kwangdong Pharmaceutical in broadening its portfolio of rare disease treatments. Elprazole is the first Fabry disease treatment in South Korea to utilize plant cell-derived recombinant proteins and is intended for long-term enzyme replacement therapy in adult patients.

Additionally, the recent recommendation for approval of a once-every-four-weeks dosing regimen by the European Medicines Agency (EMA) is seen as a strong advantage. This regimen reduces the burden on patients and healthcare providers compared to the previous biweekly administration.

The rare disease treatment market, while serving a small patient population, attracts significant interest from global pharmaceutical companies due to the high need for treatment and pricing. Particularly for enzyme replacement therapies like that for Fabry disease, there is a consistent demand, making the introduction of such treatments in South Korea directly linked to patient accessibility.

A Kwangdong Pharmaceutical representative stated, "Earlier in 2023, we signed an exclusive sales and distribution agreement for three rare drugs, including Elprazole, with Italy's Chiesi and have been progressing through the approval process. We will continue to enhance treatment accessibility by expanding the introduction of rare disease treatments."