Orsini Selected by Immedica Pharma US Inc. as Exclusive Specialty Pharmacy and Hub Services Partner for LOARGYS® (pegzilarginase-nbln)

ELK GROVE VILLAGE, Ill., March 9, 2026 /PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, is pleased to announce it was selected by Immedica Pharma US Inc. as the exclusive specialty pharmacy and hub services partner for LOARGYS^® (pegzilarginase-nbln), an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.¹ LOARGYS is the first and only therapy proven to lower arginine in patients 2 years of age and older living with ARG1-D.² See Important Safety Information below and full prescribing information for complete safety information.

ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease resulting in persistent elevation of plasma arginine, also known as hyperargininemia.³ ARG1-D affects an estimated 250 people living in the U.S. and current standard of care relies primarily on symptom management, including dietary protein restriction, arginine-free amino acid supplementation and nitrogen scavenging agents if necessary.

Through this partnership, Orsini will play a key role in helping patients, caregivers and healthcare providers navigate medication access, including insurance coverage and financial assistance. In addition to integrated specialty pharmacy and hub services, Orsini will provide 3PL services for Immedica, as previously announced.

"We're thrilled to support Immedica with comprehensive commercialization services for the launch of LOARGYS," Brandon Tom, Orsini's CEO, said. "By bringing together our 3PL capabilities, specialty pharmacy services and patient services hub, we're fully equipped to ensure patients with ARG1-D have streamlined access to this first-of-its-kind therapy. Our team is proud to stand alongside Immedica as we work to provide meaningful support for the rare disease community."

References

1. LOARGYS (pegzilarginase-nbln) US Prescribing Information. Immedica Pharma, 2026.
2. Russo RS, et al. Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled multi-centre trial. EClinicalMedicine. 2024;68:102405.
3. Burlina A, et al. Arginase 1 deficiency: a treatable form of spastic paraplegia. Neurol Sci. 2025;46:4219–4228.

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

See full prescribing information for complete boxed warning

Initiate LOARGYS in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LOARGYS, and immediately initiate appropriate medical treatment, including use of epinephrine.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions Including Anaphylaxis: Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including LOARGYS. Hypersensitivity reactions that were mild to moderate in severity occurred in 13% (6/48) of LOARGYS-treated subjects in clinical trials. Hypersensitivity reactions have included facial swelling, rash, flushing and dyspnea. The reactions generally occurred with the first few doses but may occur later in treatment.

Administration of LOARGYS should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis in a healthcare setting with appropriate medical monitoring and support measures. Premedication with an antihistamine and/or corticosteroid should be considered in patients who previously have developed a hypersensitivity reaction. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LOARGYS and immediately initiate appropriate medical treatment, including use of epinephrine. Consider the risks and benefits of re-administering LOARGYS in patients who have experienced a severe hypersensitivity reaction. Caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions and to seek immediate medical attention should symptoms occur. If a mild or moderate reaction occurs, consider treatment with antihistamines and/or corticosteroids.

ADVERSE REACTIONS

The most common adverse reactions are vomiting, pyrexia, infusion associated reactions and constipation.

USE IN SPECIFIC POPULATIONS

Pregnancy: There are no available data on LOARGYS use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.

Lactation: There is no data on the presence of LOARGYS in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for LOARGYS and any potential adverse effects on the breast-fed infant from LOARGYS or from the underlying maternal condition.

Pediatric: The safety and effectiveness of LOARGYS have been established for the reduction of plasma arginine in pediatric patients 2 years and older with ARG-1 D, in conjunction with dietary protein restriction. The safety and effectiveness of LOARGYS have not been established for the reduction of plasma arginine in pediatric patients aged less than 2 years with ARG-1 D.

Geriatric: Clinical studies of LOARGYS did not include subjects 65 years of age and older to determine whether they respond differently from younger adult subjects.

INDICATION

LOARGYS is an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.

This indication is approved under accelerated approval based on reduction of plasma arginine. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About Orsini
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services hub, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.

Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, visit www.orsini.com.

SOURCE Orsini