PR94657

BAAR, Switzerland, Feb 23, 2022, /PRNewswire=KYODO JBN/--

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 orphalabs.com

[https://www.orphalabs.com/].

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.

Contact:

Orpha Labs AG

Haldenstrasse 5

CH-6340 Baar

Switzerland

UID: CHE-209.103.038

info@orpha-labs.com

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Source: Orpha Labs AG