ToolGen gets FDA approval for TGT-001 as ODD

South Korean genome editing developer ToolGen Inc. announced on Monday that its application for orphan drug designation (ODD) of the gene correction therapy (TGT-001) for CMT1A, a peripheral nerve-related genetic disease submitted to the US Food and Drug Administration (FDA) in September is approved.

The FDA’s ODD designation supports the development and expedited approval of therapies for rare and orphan diseases with fewer than 200,000 patients.

This designation offers benefits such as tax deductions for clinical expenses, exemption from new drug application fees, and market exclusivity for seven years after approval.

Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disorder characterized by symptoms such as muscle atrophy, weakness, sensory loss, gait disturbances, and areflexia. About 40% of all CMT patients have the CMT1A subtype, caused by the duplication of the PMP22 gene, and there is currently no fundamental cure.

ToolGen's gene correction therapy TGT-001 aims to correct the root cause of CMT1A, a peripheral nerve disorder characterized by protein overexpression due to the duplication of the PMP22 gene.

The company stated that it has completed validation of the treatment strategy through animal experiments.

Write to Woo-Sang Lee at idol@hankyung.com