ABX-002 Earns Orphan Drug Designation

Release Date: 20-Sep-2021

Autobahn Therapeutics, a biotechnology company announces that US FDA has granted orphan drug designation for ABX-002, for the treatment of X-linked adrenoleukodystrophy (X-ALD). 


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.


ABX-002 acts by accumulative expression of ABCD2, a compensatory transporter which functionally complements defective ABCD1 and is a direct target gene of thyroid hormone. Through increasing thyroid hormone signaling, ABCD2 expression is increased, leading to a decrease of VLCFAs. ABX-002 also stimulates remyelination by stimulating the differentiation of oligodendrocyte precursor cells, which may be of extra benefit to AMN patients.

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