BRIEF — Raxone closer to Korean approval

Donald Trump is not the only one making progress with Korean officials this week – Santhera Pharmaceuticals has announced that the Ministry of Food and Drug Safety has accepted for review its new drug application (NDA) for Raxone (idebenone) for the treatment of Leber’s hereditary optic neuropathy (LHON) – albeit in South Korea.

The oral medication was granted orphan drug designation for LHON in South Korea, giving it up to 10 years market exclusivity from the date of approval. It is currently authorized in the European Union, Norway, Iceland, Liechtenstein and Israel at a daily dose of 900 mg for the treatment of visual impairment in adolescent and adult patients with LHON.

Raxone is the first and only medicine approved for this rare inherited disease which, if untreated, invariably leads to profound vision loss and blindness.

The NDA now submitted to the MFDS was prepared on the basis of the European marketing authorization and Santhera expects a decision from the South Korean drug regulatory authorities in around a year’s time.

Thomas Meier, chief executive of Switzerland-based Santhera, said: “The regulatory submission of Raxone for LHON in South Korea, one of the major Asian markets, underlines our geographical expansion and our commitment to providing treatment to patients worldwide, addressing a major unmet medical need in this rare disease.”