JCR Pharmaceuticals, a specialty biopharmaceutical company focused on therapies for rare and genetic diseases, has announced that Izcargo (pabinafusp alfa/JR-141) has received marketing authorisation in the United Arab Emirates (UAE) for the treatment of Hunter syndrome (Mucopolysaccharidosis Type II, MPS II).

The approval represents the first international marketing authorisation for Izcargo outside Japan, where the therapy was launched in 2021. The milestone supports JCR’s strategy to expand global access to its innovative treatments while continuing the ongoing global Phase 3 clinical development programme for the therapy.

Izcargo is the first Enzyme Replacement Therapy (ERT) developed using JCR’s proprietary J-Brain Cargo technology, which enables therapeutic enzymes to cross the Blood-Brain Barrier (BBB). This innovative approach is designed to address both the systemic and neurological manifestations of Hunter syndrome—an area that has remained a significant unmet medical need.

To support commercialisation across the region, JCR has partnered with Taiba Middle East FZ LLC (Taiba) under a distribution agreement. While JCR will pursue regulatory approvals across the Middle East and North Africa (MENA) region, Taiba will oversee the marketing, sales and distribution of Izcargo in approved markets.

Commenting on the milestone, Dr. Hiroyuki Sonoda, President and Chief Scientific Officer of JCR Pharmaceuticals, said the UAE approval marks an important step in the company’s international expansion and reflects its commitment to making innovative therapies available to more patients living with Hunter syndrome worldwide.

Dr. Saif Al Hasani, Group CEO of Taiba, noted that the approval strengthens the companies' shared commitment to improving access to advanced therapies for patients with rare diseases across the MENA region. He added that the partnership combines scientific innovation with broader accessibility to improve patient outcomes.

Hunter syndrome is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme iduronate-2-sulfatase, resulting in the accumulation of glycosaminoglycans in tissues throughout the body. The disease affects approximately 2,000–3,000 people globally and can lead to severe physical complications as well as progressive neurological impairment.

Unlike conventional enzyme replacement therapies that primarily address systemic symptoms, Izcargo incorporates JCR’s J-Brain Cargo platform to transport the therapeutic enzyme across the blood-brain barrier via transferrin receptor-mediated transcytosis. Clinical and preclinical studies have demonstrated its ability to reduce biomarkers associated with neurological disease progression while improving central nervous system outcomes.

The therapy has been commercially available in Japan since May 2021 under the brand name Izcargo IV Infusion 10 mg and is currently approved in Japan and the UAE. JCR continues to expand its global footprint while advancing innovative technologies aimed at addressing complex neurological manifestations of rare genetic disorders.