Israel’s Ministry of Health (MOH) approved the compassionate use of an investigational drug for patients with Leukocyte Adhesion Deficiency Type II (LAD-II). Orpha Labs AG announced recently that its novel experimental drug called ORL-101 will now be available under a compassionate use program for LAD-II patients under physician care. This novel formulation of an ultra-pure L-fucose currently in development is also the study drug of a Phase 3 trial to commence soon. Additionally, the U.S. Food and Drug Administration (FDA) granted a Rare Pediatric Disease Designation to ORL-101 for the treatment of patients with LAD II.
What is LAD-II?
Leukocyte Adhesion Deficiency Type II (LAD-II) Is an autosomal recessive primary immunodeficiency characterized by impaired leukocyte motility and moderate to severe neurodevelopmental retardation. The genetic defect in LAD-II patients has been shown to be various mutations in the SLC35C1 gene which encodes for GDP-Fucose Transporter 1. This transporter mediates GDP-Fucose uptake into Golgi vesicles, and its dysfunction results in the absence of fucosylated glycans on the membranes of cells, leading to the loss of E- and P-selectin ligands on leukocytes, thus resulting in an inability of circulating leukocytes to efficiently migrate to the sites of infection, which, in turn, causes persistent leukocytosis and recurrent episodes of life-threatening infections.
Orpha Labs AG
Based in Switzerland, Orpha Labs AG describes itself as a patients’ needs-driven research and development company committed to discovering, developing, and delivering effective drugs for neglected ultra-rare diseases. Founded by Alp Bugra Basat, MD, the company’s Rare Pediatric Disease Designation of ORL-101 is also associated with what the FDA refers to as a Priority Review Voucher” (PRV). This means that assuming that ORL-101 makes it successfully through Phase 3 clinical trials and the company is able to secure an FDA approved New Drug Application (NDA) they could benefit as well from additional incentives from the world’s apex drug regulatory body.