RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Orphan Drug (Australia)

Structure/Sequence

Sequence Code 523960959

Source: *****

Clinical Trials associated with Pegzilarginase

CTIS2024-510797-25-00

/ CompletedPhase 3

A Phase 3 Open-Label Study of Safety, Pharmacokinetics and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency

Start Date18 Dec 2024

Sponsor / Collaborator

Immedica Pharma AB

NCT06582524

/ CompletedPhase 3

A Phase 3 Open-Label Study of Safety, Pharmacokinetics, and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

This is an open-label, multicentre study to evaluate the safety, PK, and activity (PD) of weekly subcutaneous (SC) administration of pegzilarginase in subjects with ARG1-D who are < 24 months of age. The study consists of a screening period of up to 4 weeks, a subsequent 12-week treatment period, and a safety follow-up period of 8 weeks.

Start Date30 Aug 2024

Sponsor / Collaborator

Immedica Pharma AB

NCT05676853

/ TerminatedPhase 3

A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency

This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.

Start Date04 Apr 2023

Sponsor / Collaborator

Spyre Therapeutics, Inc.

100 Clinical Results associated with Pegzilarginase

100 Translational Medicine associated with Pegzilarginase

100 Patents (Medical) associated with Pegzilarginase

Literatures (Medical) associated with Pegzilarginase

01 Sep 2025·JOURNAL OF INHERITED METABOLIC DISEASE

Correction to “Long‐Term Efficacy and Tolerability of Pegzilarginase in Arginase 1 Deficiency: Results of Two International Multicentre Open‐Label Extension Studies”

01 Sep 2025·NEUROLOGICAL SCIENCES

Arginase 1 deficiency: a treatable form of spastic paraplegia

Review

Author: Battini, Roberta ; Gasperini, Serena ; Porta, Francesco ; Vici, Carlo Dionisi ; Burlina, Alberto ; Ardissone, Anna ; Pession, Andrea ; Burlina, Alessandro

Abstract:

Background:

Arginase 1 deficiency (ARG1-D) is a rare hereditary urea cycle disorder characterized by elevated arginine levels, resulting in progressive neurological impairment and severe physical and cognitive disability. Due to its low prevalence, overlapping symptoms with other neurological disorders, and current limitations in newborn screening tools, ARG1-D is often misdiagnosed or diagnosed late, limiting access to early interventions.

Aim:

This review and expert opinion aim to provide an overview of the clinical manifestations, diagnostic challenges, and treatment options for ARG1-D, offering a practical resource for specialists to recognize this rare, progressive, yet treatable disease.

Results:

ARG1-D typically presents with progressive spastic paraplegia, developmental delays, cognitive impairment, and seizures, with symptom onset and severity varying by age. Differential diagnoses mainly include hereditary spastic paraplegia, cerebral palsy, and hyperornithinemia-hyperammonemia-homocitrullinuria syndrome, each with distinct clinical features and biochemical markers. Potential red flags for ARG1-D include elevated plasma arginine levels, spasticity, seizures, and cognitive impairment. These should prompt further examinations to confirm the diagnosis, which is based on biochemical assays for hyperargininemia and on genetic testing. Once confirmed, early treatment is advised, including dietary protein restriction, ammonia scavengers, antiepileptic drugs, and novel therapies, such as pegzilarginase, which targets the disease’s metabolic root.

Conclusion:

Experts stress the importance of increased awareness of ARG1-D characteristics, noting that early recognition and treatment are crucial to patient outcomes. Greater recognition of ARG1-D’s distinctive features, differential diagnosis, and diagnostic tools, even among non-specialist clinicians, could help prevent misdiagnoses and facilitate the identification of this rare yet treatable condition.

01 Jul 2025·JOURNAL OF INHERITED METABOLIC DISEASE

Long‐Term Efficacy and Tolerability of Pegzilarginase in Arginase 1 Deficiency: Results of Two International Multicentre Open‐Label Extension Studies

Article

Author: McNutt, Markey ; Batzios, Spyros ; Russo, Rossana Sanchez ; Rudebeck, Mattias ; Ganesh, Jaya ; Rutsch, Frank ; Gasperini, Serena ; Schulze, Andreas ; Enns, Gregory M. ; Teles, Elisa Leão ; Brassier, Anaïs

ABSTRACT:

Arginase 1 deficiency (ARG1‐D) is an autosomal recessive urea cycle disorder characterised by chronic hyperargininaemia, progressive spasticity, loss of mobility, and cognitive dysfunction. Standard of care (SOC), based on dietary protein restriction, rarely prevents progression. Pegzilarginase, a recombinant human enzyme, is the first approved disease‐modifying therapy. We report outcomes from Study 102A (n = 14; up to 5 years) and the PEACE long‐term extension (LTE) (n = 31; up to 3 years). Weekly pegzilarginase was administered with SOC. Outcomes included functional mobility (2‐/6‐minute walk tests [2MWT/6MWT], Gross Motor Function Measure [GMFM] D/E), spasticity (Modified Ashworth Scale [MAS]), plasma arginine, guanidino compounds, and safety. In PEACE, LTE arms were named according to initial 24‐week double‐blind treatment: placebo–pegzilarginase or pegzilarginase–pegzilarginase. Of 39 evaluable participants, 37 (95%) met composite response or achieved maximum score in ≥ 1 motor function domain. In 102A, mean 6MWT improved to 68.2 m (+19%; n = 12); GMFM‐D/E increased by 2.7/3.7. In PEACE (pegzilarginase–pegzilarginase; placebo–pegzilarginase), 2MWT improved to 16.5 m (+25%; n = 6) and 13.5 m (+16%; n = 2); GMFM‐D/E improved by 4.3/6.0 (n = 6) and 5.3/11.3 (n = 3). Spasticity improved in 21/25 (84%), with 12 reaching MAS 0 (no spasticity). Pegzilarginase sustained plasma arginine control: in 102A, means were 118 μmol/L at Week 192 (n = 9); in PEACE, < 115 μmol/L through Week 96 (n = 11). Guanidinoacetic acid normalised; N‐acetylarginine approached normal; argininic acid and α‐keto‐δ‐guanidinovaleric acid declined by > 50%. Most adverse events were mild/moderate; no treatment‐related discontinuations or persistent antibodies occurred. Pegzilarginase produced sustained improvements in mobility, spasticity and biochemical control, supporting early intervention, long‐term use and disease modification in ARG1‐D.

News (Medical) associated with Pegzilarginase

10 Dec 2025

·www.immedica.com

Loargys® (pegzilarginase) granted marketing authorization in Oman for the treatment of arginase 1 deficiency (ARG1-D)

These cookies allow the website to remember choices you make and provide enhanced, more personal features. The information these cookies collect may be anonymized and they cannot track your browsing activity on other websites. These cookies allow us to recognise and count the number of visitors and to see how visitors move around our website when they are using it. This helps us to improve the way our website works, for example, by ensuring that users are finding what they are looking for easily. These cookies enable the website to provide enhanced functionality and personalisation. They may be set by us or by third party providers whose services we have added to our pages. If you do not allow these cookies then some or all of these services may not function properly. Stockholm, Sweden, December 10, 2025 – Immedica today announces that Loargys® (pegzilarginase) has been granted marketing authorization in Oman for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. ARG1-D is a rare, inherited metabolic disorder that can lead to the accumulation of arginine and associated toxic metabolites, with potentially serious, progressive and life-impacting consequences for patients and families. “The approval in Oman is the first in the MENA region and supports Immedica’s ambition to broaden access to essential therapies for rare diseases in markets where unmet medical need remains high” said Ashraf Attia General Manager of Immedica in MENA. ----------- About Loargys® Loargys (pegzilarginase) is a novel recombinant human enzyme and has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma accompanied by improvements in clinical outcomes. Loargys is approved in the EU, UK and Oman for the treatment of ARG1-D, also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. It is the first and only disease-modifying treatment for ARG1-D. About ARG1-D ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares overlapping features with other UCDs and the most prominent is the impairment in excreting nitrogen. However, in ARG1-D, hyperammonemia is generally less severe and instead these patients show spasticity, which other subtypes do not. The principal defect in ARG1-D leads to accumulation of plasma arginine and its toxic metabolites, which occurs in almost all patients with this disorder. Patients are often diagnosed in late infancy or early childhood and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, 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 and specialty care. Immedica was founded in 2018 and employs today around 150 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.immedica.com. Immedica contact: Linda Holmström VP, Head of Communication linda.holmstrom@immedica.com Press release - Loargys approved in Oman Follow us to stay up to date with what is happening at Immedica. We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Drug ApprovalPhase 2

09 Dec 2025

·www.immedica.com

Immedica submits pegzilarginase application to the Australian Therapeutic Goods Administration (TGA)

Stockholm, Sweden, December 9, 2025 - Immedica today announced that it has submitted a prescription medicine application for pegzilarginase to the Australian Therapeutic Goods Administration (TGA). The TGA has issued a Milestone 2 Notification Letter confirming that the application has successfully passed preliminary assessment under the Therapeutic Goods Act 1989. The application is now considered effective and has been formally accepted for evaluation, representing the next step toward potential inclusion of pegzilarginase in the Australian Register of Therapeutic Goods (ARTG). "We are pleased to have achieved this milestone with the TGA," said Anders Edvell, CEO. "The acceptance of our application for evaluation is an important step in making pegzilarginase available to patients in Australia living with this severe and ultra-rare condition." About pegzilarginase Pegzilarginase is a novel recombinant human enzyme and has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma accompanied by improvements in clinical outcomes. About ARG1-D ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares overlapping features with other UCDs and the most prominent is the impairment in excreting nitrogen. However, in ARG1-D, hyperammonemia is generally less severe and instead these patients show spasticity, which other subtypes do not. The principal defect in ARG1-D leads to accumulation of plasma arginine and its toxic metabolites, which occurs in almost all patients with this disorder. Patients are often diagnosed in late infancy or early childhood and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, 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 and specialty care. Immedica was founded in 2018 and employs today around 160 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.immedica.com. Immedica contact: Linda Holmström VP, Head of HR & Communication linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

31 Oct 2025

·www.immedica.com

Immedica submits New Drug Submission (NDS) for Loargys® (pegzilarginase) to Health Canada

Stockholm, Sweden, October 31, 2025 – Immedica today announced that the submitted New Drug Submission (NDS) for Loargys® (pegzilarginase) to Health Canada has been accepted for review. This milestone marks the company’s first regulatory filing in Canada, highlighting Immedica’s continued expansion and commitment to addressing rare diseases on a global scale. The pegzilarginase NDS has been granted priority review by Health Canada, reflecting the therapy’s potential to address a significant unmet medical need. "The submission of pegzilarginase to Health Canada represents an important step in our mission to bring transformative therapies to patients with rare diseases worldwide," said Anders Edvell, CEO of Immedica. "This achievement reflects the dedication and expertise of our team, and we look forward to collaborating with Health Canada throughout the review process." About Loargys® Loargys (pegzilarginase) is a novel recombinant human enzyme and has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma accompanied by improvements in clinical outcomes. It is the first and only disease-modifying treatment. About ARG1-D ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares overlapping features with other UCDs and the most prominent is the impairment in excreting nitrogen. However, in ARG1-D, hyperammonemia is generally less severe and instead these patients show spasticity, which other subtypes do not. The principal defect in ARG1-D leads to accumulation of plasma arginine and its toxic metabolites, which occurs in almost all patients with this disorder. Patients are often diagnosed in late infancy or early childhood, and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, 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 and specialty care. Immedica was founded in 2018 and employs today around 150 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.immedica.com. Immedica contact: Linda Holmström VP, Head of HR & Communication linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Priority Review

100 Deals associated with Pegzilarginase

External Link

KEGG	Wiki	ATC	Drug Bank
D11695	-	-	DB14780

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperargininemia	European Union	Immedica Pharma AB	15 Dec 2023
Hyperargininemia	Iceland	Immedica Pharma AB	15 Dec 2023
Hyperargininemia	Liechtenstein	Immedica Pharma AB	15 Dec 2023
Hyperargininemia	Norway	Immedica Pharma AB	15 Dec 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Extensive stage Small Cell Lung Cancer	Phase 2	United States	Spyre Therapeutics, Inc.	21 Dec 2017
Extensive stage Small Cell Lung Cancer	Phase 2	Puerto Rico	Spyre Therapeutics, Inc.	21 Dec 2017
Refractory acute myeloid leukemia	Phase 2	United States	Spyre Therapeutics, Inc.	01 Aug 2016
Refractory acute myeloid leukemia	Phase 2	Canada	Spyre Therapeutics, Inc.	01 Aug 2016
Refractory Myelodysplastic Syndrome	Phase 2	United States	Spyre Therapeutics, Inc.	01 Aug 2016
Refractory Myelodysplastic Syndrome	Phase 2	Canada	Spyre Therapeutics, Inc.	01 Aug 2016
Relapsing acute myeloid leukemia	Phase 2	United States	Spyre Therapeutics, Inc.	01 Aug 2016
Relapsing acute myeloid leukemia	Phase 2	Canada	Spyre Therapeutics, Inc.	01 Aug 2016
Advanced Malignant Solid Neoplasm	Phase 2	United States	Spyre Therapeutics, Inc.	01 Oct 2015
Uveal Melanoma	Phase 2	United States	Spyre Therapeutics, Inc.	01 Oct 2015

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03921541 (PEACE, CTgov)	Phase 3	Hyperargininemia	32	Pegzilarginase (Pegzilarginase)	vxicctjvzp(knlzzbtunt) = kplvhsblvb tjywqjhjjs (qzftmxgxld, 1.635) View more	-	19 Nov 2024
NCT03921541 (PEACE, CTgov)	Phase 3	Hyperargininemia	32	Placebo (Placebo)	vxicctjvzp(knlzzbtunt) = skmvkedjkd tjywqjhjjs (qzftmxgxld, 1.371) View more	-	19 Nov 2024
NCT03921541 (PEACE, SSIEM2022)	Phase 3	Hyperargininemia plasma arginine (pArg)	32	Pegzilarginase	cunguqrebv(ovakadyslp) = bliivhnpav nbrjgpfvnn (lgbaeinxpi )	Positive	30 Aug 2022
NCT02488044 \| NCT03378531 (Pubmed \| J Inherit Metab Dis) Manual	Phase 1/2	Hyperargininemia	16	pegzilarginase	ahxnxrazgw(bgzoyubsac) = five patients were all observed in 101A snvipxacxn (ojnfvkqhsf ) View more	Positive	01 Jul 2021
SSIEM2019	Phase 1/2	Hyperargininemia plasma arginine	16	Pegzilarginase	vbttgqksay(vsunncvszm) = taxrcyzlgo zmteskqcbs (ofiyaeaunr )	-	25 Aug 2019
SITC2018	Phase 1	Neoplasms	-	Pegzilarginase	kgcxdsknvq(yzimxqpipn) = increased levels of granzyme A, IFN-g and TNF-a hwimlovldp (chypcsjlaa ) View more	Positive	06 Nov 2018
SITC2018	Phase 1	Neoplasms	-	aOX40 mAb		Positive	06 Nov 2018
NCT02561234 (AACR 12018 \| AACR 22018) Manual	Phase 1	HR-positive/HER2-low Solid Tumors	40	pegzilarginase	tfrwrioiky(dduwlpufgn) = 0.33 mg/kg once weekly cfmcodzuxy (onpbrmgaas ) View more	Positive	01 Jul 2018
NCT02732184 (ASCO 12018 \| ASCO 22018) Manual	Phase 1	Refractory acute myeloid leukemia \| Myelodysplastic Syndromes	21	pegzilarginase	rrtnagpoky(thwsjdwkrp) = hlwsmzuhxk hbgywkiubi (akgivccjzk )	Positive	04 Jun 2018

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