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Orpha Labs AG Announces the Initiation of a Phase III Trial Evaluating ORL-101 for Treatment of Leukocyte Adhesion Deficiency Type II

BAAR, Switzerland, Feb 23

Orpha Labs AG, a clinical-stage biopharmaceutical company focused on diseases with unmet needs, today announced the initiation of the Phase III trial with ORL-101 in patients with Leukocyte Adhesion Deficiency Type II (“LAD-II”). Orpha Labs AG is working with investigators to switch patients from a compassionate use study initiated last year to this Phase III study.

“The data obtained from the compassionate use program was an invaluable guide for our late-stage clinical development activities. We look forward to enrolling LAD-II patients in our Phase III study and initiating a rolling submission of the NDA in the United States.” said Dr. Alp Bugra Basat, Founder and Chief Executive Officer of Orpha Labs AG. “A very important step for LAD-II patients and their caregivers. I am very excited about this development” said Dr. Amos Etzioni, Professor Emeritus of Pediatrics and Immunology at The Rappaport Faculty of Medicine and Chair of Scientific Advisory Board at Orpha Labs AG.

Orpha Labs AG has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for ORL-101 which is under investigation for the treatment LAD II. If a New Drug Application (NDA) is approved for ORL-101 treatment of LAD-II, Orpha Labs AG is eligible to receive a Priority Review Voucher (PRV) from the FDA. A PRV can be redeemed to obtain priority review for any subsequent marketing application. In addition, the FDA has granted an Orphan Drug Designation to ORL-101 for this indication, which will provide seven (7) years of marketing exclusivity upon approval. Orpha Labs AG has also received a fast-track designation from the FDA, which may accelerate the development and approval process of ORL-101 for the treatment of LAD-II.

For more information on the Phase III trial, visit [].

About ORL-101
ORL-101 is an investigational pharmaceutical-grade L-fucose manufactured according to the Current Good Manufacturing Practice (cGMP). ORL-101 is believed to act by improving the fucosylation of various plasma membrane glycoproteins including E- and P-selectin ligands.

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 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
UID: CHE-209.103.038

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Source: Orpha Labs AG