Novel Therapy for Heparin Induced Thrombocytopenia Gets Orphan Drug Status

The Food and Drug Administration (FDA) has granted Orphan Drug designation to VLX-1005 (Veralox Therapeutics) for the treatment of heparin induced thrombocytopenia (HIT).

VLX-1005 is a first-in-class and selective small molecule inhibitor of 12-lipoxygenase, a key target within the arachidonic acid pathway. In preclinical studies, VLX-1005 has been shown to halt immune driven platelet activation and thrombosis.

“We believe VLX-1005, with its novel mechanism of action, has great potential to be the first disease modifying therapy for HIT in over 20 years,” said Matthew B. Boxer, PhD, Chief Operating Officer of Veralox Therapeutics.

Currently approved treatments for HIT include the direct thrombin inhibitors argatroban, bivalirudin, and lepirudin.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

Reference

Veralox Therapeutics announces FDA Orphan Drug designation for VLX-1005. [press release]. Frederick, MD: Veralox Therapeutics; January 26, 2021.