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Brand Name
Loargys
Generic Name
Pegzilarginase-Nbln
View Brand Information FDA approval date: February 23, 2026
Form: Injection
What is Loargys (Pegzilarginase-Nbln)?
LOARGYS is indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency , 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. 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 , 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.
Approved To Treat
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Related Clinical Trials
A Multicenter, Prospective Study Assessing the Safety of Loargys Arginine Test System for Determining Arginine in Loargys-treated Patients
Summary: Arginase 1 deficiency (ARG1-D) is a rare condition in which the body cannot properly break down a substance called arginine. This leads to high levels of arginine and related substances in the blood, which can cause serious health problems and reduce quality of life. Loargys is a new treatment designed to lower arginine levels in people with ARG1-D. It works by providing a modified version of a na...
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Brand Information
Loargys (pegzilarginase-nbln)
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
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.
Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur
1INDICATIONS AND USAGE
LOARGYS is 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
2DOSAGE FORMS AND STRENGTHS
LOARGYS (pegzilarginase-nbln) is a clear to slightly opalescent, colorless to slightly yellow or slightly pink solution.
Injection: 2 mg/0.4 mL or 5 mg/mL in a single-dose vial.
3CONTRAINDICATIONS
None.
4ADVERSE REACTIONS
The following adverse reactions are described below and elsewhere in the labeling:
- Hypersensitivity Reactions
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety of LOARGYS was evaluated in a randomized, double-blind, placebo-controlled trial in pediatric and adult patients with ARG1-D (Trial 1). Additional safety information was derived from Trial 2, a Phase 1 open-label trial that evaluated 16 patients between the ages of 5 to 31 years to assess safety, PK and PD of LOARGYS, and Trial 3, an open-label extension including 14 patients from Trial 2.
Adverse Reactions from Trial 1 (Double-Blind Period)
A total of 21 patients between the ages of 2 and 28 years of age, at enrollment, received intravenous LOARGYS dosages up to 0.2 mg/kg once weekly and 11 patients received placebo for 24 weeks
Table 1 summarizes the adverse reactions reported in ≥ 2 LOARGYS-treated patients and at a higher incidence in LOARGYS-treated patients compared to placebo-treated patients in Trial 1.
Description of Selected Adverse Reactions
Hypersensitivity Reactions: Hypersensitivity reactions with symptoms including facial swelling, rash, flushing and dyspnea were reported in 13% (6/48) of LOARGYS-treated patients during clinical trials [see Warnings and Precautions (.
Injection Site Reactions: Injection site reactions were reported in 14% (6/44) of patients after subcutaneous LOARGYS administration during the open-label extension periods in Trial 1 and Trial 3. Signs and symptoms included pain, erythema, swelling, irritation, and rash at the injection site.
Immunogenicity: Anti-Drug Antibody-Associated Adverse Reactions
Across Trials 1, 2, and 3, in patients with ARG1-D, the incidence of hypersensitivity reactions was 42% (5/12) in LOARGYS-treated patients who developed anti-drug antibodies (ADA) (i.e., anti-pegzilarginase-nbln antibodies and/or anti-PEG antibodies) and 3% (1/36) in those who were ADA-negative
5DESCRIPTION
Pegzilarginase-nbln is a trimeric cobalt substituted recombinant human arginase 1 enzyme conjugated with six to twelve moles of 5 kDa monomethoxy polyethylene glycol (mPEG) per monomer. The molecular weight of pegzilarginase-nbln is approximately 224 to 344 kDa.
LOARGYS (pegzilarginase-nbln) injection is a sterile, preservative-free, clear to slightly opalescent, and colorless to slightly yellow or slightly pink solution in a single-dose vial for intravenous infusion or subcutaneous injection. Available as 2 mg of pegzilarginase-nbln in 0.4 mL or 5 mg of pegzilarginase-nbln in 1 mL.
Each 1 mL contains 5 mg pegzilarginase-nbln, dibasic potassium phosphate (0.7 mg), glycerin (15 mg), monobasic potassium phosphate (0.14 mg), sodium chloride (2.92 mg), Water for Injection, USP, and hydrochloric acid and/ or sodium hydroxide to adjust pH to 7 to 7.6 The resultant concentration is 5 mg/mL.
6CLINICAL STUDIES
The effectiveness of LOARGYS for the treatment of hyperargininemia in adult and pediatric patients with ARG1-D was evaluated in Trial 1 (NCT03921541) which was a multicenter, double-blind, 24-week placebo-controlled trial with a long-term open-label extension period of up to 150 weeks. A total of 32 patients were randomized 2:1 to receive intravenous LOARGYS (n=21) or placebo (n=11) once weekly for 24 weeks.
Of the 32 patients randomized in Trial 1, 19 were males (59%) and 13 were females (41%). Patients were between 2 to 29 years of age with 90% of patients between 2 to 17 years of age (median pediatric age was 9 years) at enrollment. The patient population consisted of 14 Whites (44%), 6 Asians (19%), 2 Black or African Americans (6%), and 2 in mixed racial groups (6%). Ethnicity consisted of 9 patients who were Hispanic or Latino (28%) and 23 who were Not Hispanic or Latino (72%).
LOARGYS-treated patients received an initial dosage of 0.1 mg/kg and were titrated within a range of 0.05 mg to 0.2 mg/kg, as clinically indicated. Patients who previously had an ARG1-D-related dietary regimen or ammonia scavengers continued to receive these treatment(s) throughout the initial trial period. A total of 31 patients completed the double-blind period; one patient in the LOARGYS arm discontinued for personal reasons.
The primary endpoint in Trial 1 was the change from baseline in plasma arginine at Week 24.
LOARGYS-treated patients had a significant mean reduction in plasma arginine levels from baseline to Week 24 (
All 31 patients (n=20 LOARGYS and n=11 placebo) who completed the double-blind period entered the extension period and received open label treatment of LOARGYS:
- First 8 weeks: Patients who were previously treated with intravenous LOARGYS continued their weekly intravenous LOARGYS dosage and patients who were previously treated with placebo initially received 0.1 mg/kg of weekly intravenous LOARGYS. The LOARGYS dosage in both groups could be modified based on plasma arginine levels.
- After the first 8 weeks: Patients had the option to receive weekly subcutaneous LOARGYS (same as the intravenous dosage) that could be modified based on plasma arginine levels up to a maximum of 0.2 mg/kg weekly.
The median duration of LOARGYS exposure in the extension period, excluding the double-blind period of 24 weeks, was 94 weeks (range: 62 to 152 weeks). During the extension period, patients who switched from placebo to LOARGYS achieved similar reductions in mean plasma arginine levels as those who had received LOARGYS from the start of the study (
Notes: 95% confidence interval of mean (pre-dose) is displayed; Recommended plasma arginine levels: below 200 micromolar; Normal range defined as 40–115 micromolar in Trial 1. The x-axis unit of time is visit week.
7HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
LOARGYS (pegzilarginase-nbln) is supplied as a sterile, preservative-free, clear to slightly opalescent and colorless to slightly yellow or slightly pink solution. LOARGYS is available as:
- One single-dose vial 2 mg/0.4 mL in a carton (NDC 81583-102-01)
- One single-dose vial 5 mg/mL in a carton (NDC 81583-105-01).
Storage and Handling
Store refrigerated at 2 °C to 8 °C (36 °F to 46 °F) in the original carton to protect from light. Do not freeze. Do not shake.
8PATIENT COUNSELING INFORMATION
Hypersensitivity Reactions Including Anaphylaxis:
Advise the patient and caregiver that life-threatening hypersensitivity reactions, including anaphylaxis may occur with LOARGYS treatment.
Advise the patient or caregiver that anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Inform the patient and caregiver of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis, and to seek immediate medical care should symptoms occur
9PRINCIPAL DISPLAY PANEL - 2 mg/0.4 mL Carton
NDC: 81583-102-01
Rx only
LoargysInjection
2 mg/0.4 mL
For Intravenous Infusion After
One 0.4 mL single-dose vial
Immedica
10PRINCIPAL DISPLAY PANEL - 5 mg/1 mL Carton
NDC: 81583-105-01
Rx only
Loargys
5 mg/mL
For Intravenous Infusion After
One 1 mL single-dose vial
Immedica