FDA approves Chinese eye cancer treatment for development support in the US

Designation will ease the gene-based breakthrough’s transition from laboratory to clinic with tax breaks and other investment incentives

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The US Food and Drug Administration has granted orphan drug designation to a promising genetic-based treatment developed in China that targets uveal melanoma. Photo: AP

Published: 9:00am, 3 Jan 2025

A breakthrough treatment developed by a group of Chinese researchers to treat a rare but deadly eye cancer has been granted a special status in the US that will “greatly facilitate” its transition from laboratory to clinic.

Known as an aptamer drug conjugate (ApDC), the next generation gene-based treatment is the first of its kind in the world to be granted orphan drug designation by the US Food and Drug Administration (FDA).

The medication was jointly developed by researchers at the Hangzhou Institute of Medicine (HIM), an affiliate of the Chinese Academy of Sciences, and Wenzhou Medical University.

The FDA grants orphan status to medicines and biologics for rare diseases that meet certain criteria, opening the way for incentives such as accelerated approval and tax breaks to support their development.

Announcing the FDA’s decision on Saturday, the HIM said the treatment has the potential to bring new hope to patients around the world with uveal melanoma, which affects around 2,500 people in the US each year.

Because the early symptoms of the disease are often not obvious, many patients are not diagnosed until it has reached an advanced stage.