Loargys® (pegzilarginase-nbln) now available to patients in the U.S. just 5 weeks after FDA approval
CHICAGO, Illinois -April 2, 2026- Immedica Pharma US Inc. announced today that Loargys® (pegzilarginase-nbln) is now commercially available to patients two years and older with Arginase 1 Deficiency (ARG1-D) for the treatment of hyperarginemia, just over five weeks after receiving accelerated approval from the U.S. Food and Drug Administration (FDA) on February 23, 2026. This launch represents one of the fastest transitions from regulatory approval to patient availability in the company’s history.
“Getting a medicine approved is a significant milestone” said Dan Camardo, President of Immedica Pharma US Inc. “We began preparing for this launch long before approval day because we believe that every day a patient waits for a needed treatment, is a day too long.”
LOARGYS is available through two distribution channels: Orsini Specialty Pharmacy, the exclusive specialty pharmacy for LOARGYS, and Morris & Dickson, the exclusive specialty distributor. Immedica Pharma has coordinated closely with both partners to help ensure timely patient access to therapy.
Immedica Pharma has also launched There for Rare, a patient support program providing nonmedical education to help eligible individuals prescribed LOARGYS navigate each stage of the treatment journey, including financial assistance options. To begin the enrollment process, visit www.loargys.com, navigate to the “Resources” tab, and download and submit a Patient Enrollment Form.
About ARG1-D
ARG1-D is an ultra-rare and serious inherited metabolic disorder. The principal defect in ARG1-D leads to accumulation of plasma arginine (hyperargininemia,) and its toxic metabolites. Patients are often diagnosed in late infancy or early childhood, and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality. ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares some overlapping features with other UCDs, including impaired nitrogen excretion. However, in ARG1-D, hyperammonemia is generally less severe.
About Loargys® (pegzilarginase-nbln)
LOARGYS is a novel, recombinant, human arginase-1 enzyme that has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma, making it the first and only therapy proven to lower plasma arginine. LOARGYS is approved 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.
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.
Please see full Prescribing Information for LOARGYS.
About Immedica Pharma US Inc.
Immedica Pharma US Inc. is an affiliate of Immedica Pharma AB, a pharmaceutical company, headquartered in Stockholm, Sweden. Immedica is focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE endocrinology, and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East, and the United States. Immedica is backed by the investment firms KKR and Impilo.
For more information visit www.ImmedicaUS.com.
About Morris & Dickson
Morris & Dickson is the industry’s largest independent full-line and specialty pharmaceutical distributor with a singular focus on reliable, next-day delivery of drugs and related products to health systems, independent and specialty pharmacies, specialty clinics and infusion centers, and alternative care facilities. Serving pharmacies since 1841, Morris & Dickson continues to grow to meet customers’ needs with a comprehensive inventory of over 35,000 SKUs and a state-of-the-art, 12-acre automated distribution center, located in Louisiana. M&D plans to open a second distribution center designed to support its national and specialty growth in early 2026, located in Olive Branch, MS. www.morrisdickson.com
Immedica contact:
Linda Holmström
VP, Head of HR & Communication
linda.holmstrom@immedica.com
For M&D Media inquiries:
Cassandra Johnson
SVP, Marketing & Communications
CJohnson@morrisdickson.com
214-288-5471
