PR87969

BAAR, Switzerland, Feb. 8, 2021 /PRNewswire=KYODO JBN/--

-- First Patient to Receive ORL-101 in Israel This Week

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.

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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

SOURCE: Orpha Labs AG