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 -- 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 OfficerAlp 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.

AboutOrpha 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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