First patient to receive first Orpha Labs drug this week

Orpha Labs is committed to discovering and developing treatments for ultra-rare diseases. One of these diseases is Leukocyte Adhesion Deficiency Type II. According to Orphanet LAD II is characterized by recurrent bacterial infections, severe growth delay and severe intellectual deficit. The genetic defect in LAD-II patients has been shown to be various mutations in the "SLC35C1" gene.

Orpha Labs has now made ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) under its compassionate use program. 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. The FDA has also granted a Rare Pediatric Disease Designation to ORL-101 for the treatment of patients with LAD II. 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."

Orpha Labs was incorporated in Switzerland in 2019. Founder Alp Bugra Basat received his M.D. degree from Hacettepe University of Ankara, Turkey, and worked in several laboratories and clinics at Harvard Medical School and University of Oxford. He also worked as an associate at Rhea Capital Partners.