Orpha Labs AG Initiates Compassionate Use Program for ORL-101 in Leukocyte Adhesion Deficiency Type II (LAD-II)

First Patient to Receive ORL-101 in Israel This Week

BAAR, Switzerland, Feb. 8, 2021 /PRNewswire/ -- Orpha Labs AG today announced its compassionate use program to make ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) patients. ORL-101 is a novel formulation of an ultra-pure L-fucose currently in development for the treatment of LAD II patients.

The Israeli Ministry of Health (MOH) approved the use of ORL-101 for LAD-II patients under Orpha Labs' compassionate use program. Orpha Labs expects to enroll LAD-II patients in a Phase III trial soon.

Orpha Labs' Founder and Chief Executive Officer Alp Bugra Basat, MD, said, "We are proud to announce this development. ORL-101 is the first product authorized for compassionate use in a program for LAD-II patients, an important step in our work to bring products into clinical development to help patients with ultra-rare disorders."

The FDA has granted a Rare Pediatric Disease Designation to ORL-101 for the treatment of patients with LAD II. If a new drug application ("NDA") for ORL-101 for patients with LAD II is approved, the Company may be eligible to receive a Priority Review Voucher ("PRV") from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application.

About Leukocyte Adhesion Deficiency Type II (LAD-II)
LAD-II (OMIM # 266265) 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.

About Orpha Labs AG
Orpha Labs AG is a patients' needs-driven research and development company committed to discovering, developing, and delivering effective drugs for neglected ultra-rare diseases. Our mission is to provide innovative products that improve not only the survival rates but also the quality of life for these patient populations.

Orpha Labs AG
Haldenstrasse 5
CH-6340 Baar
Switzerland
UID: CHE-209.103.038

Alp Bugra Basat, MD
Chief Executive Officer
info@orpha-labs.com
+41 75 418 88 29

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