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

Structure/Sequence

Sequence Code 523960959

Source: *****

Clinical Trials associated with Pegzilarginase

NL-OMON58219

/ SuspendedNot Applicable

Analysis of patients with arginase 1 deficiency treated with Loargys in standard clinical care enrolled in a European, non-interventional, multicentre registry - Analysis of standard clinical care treatment of ARG1 deficient patients enrolled in European multicentre registry

Start Date01 Feb 2026

Sponsor / Collaborator

Immedica Pharma AB

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

100 Clinical Results associated with Pegzilarginase

100 Translational Medicine associated with Pegzilarginase

100 Patents (Medical) associated with Pegzilarginase

Literatures (Medical) associated with Pegzilarginase

01 Mar 2026·JOURNAL OF INHERITED METABOLIC DISEASE

Clinical Characteristics of Arginase 1 Deficiency: Natural History Insights From International Clinical Trials

Article

Author: Enns, Gregory M. ; Sharma, Reena ; Karall, Daniela ; Ghosh, Arunabha ; Shelkowitz, Emily ; Rudebeck, Mattias ; Gorce, Magali ; Zori, Roberto ; Braverman, Nancy ; McNutt, Markey ; Chang, Richard

ABSTRACT:

Arginase 1 deficiency (ARG1‐D) is an ultra‐rare inherited metabolic disorder of the urea cycle, caused by partial or complete loss of arginase 1 function, characterised by hyperargininaemia and a distinct, progressive neurological phenotype. The clinical development programme of pegzilarginase, a recombinant human ARG1 enzyme therapy, provides an opportunity to study the largest ARG1‐D cohort to date. The analysis included 48 paediatric and adult subjects (≥ 2 years) enrolled in the pegzilarginase trials. Baseline data collected before treatment included demographics, genotypes, red blood cell arginase activity, biochemical measures, age of symptom onset, neuromotor and neurological characteristics, growth indicators, quality of life, and use of treatments and assistive devices. The mean (SD) age of onset was 2.2 (3.6) years, which preceded diagnosis at 3.7 (5.0) years. Clinical features included motor impairment (48/48, 100%), spasticity (33/48, 69%), cognitive deficits (31/48, 65%), intellectual disability (23/36, 64%), speech and language deficits (26/48, 54%), and seizures (18/48, 38%), with symptom‐onset data consistent with a progressive phenotype. Median GMFCS Level II indicated moderate mobility limitation; two‐thirds scored < 69 on FSIQ, and mean PedsQL total proxy scores were around 20% lower than typically developing peers. All subjects followed a protein‐restricted diet, and 90% used ammonia scavengers. ARG1‐D presents with a heterogeneous array of progressive and debilitating neurologic symptoms. These findings reflect the progressive impact of the disease and offer insights into its burden and natural history based on a large cohort, assessed using standardised neuromotor, cognitive, and quality‐of‐life instruments across international sites.

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 ; Pession, Andrea ; Ardissone, Anna ; 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.

News (Medical) associated with Pegzilarginase

09 Mar 2026

·www.prnewswire.com

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 LOARGYS (pegzilarginase-nbln) US Prescribing Information. Immedica Pharma, 2026. 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. 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 . SOURCE Orsini 21% more press release views with Request a Demo

Phase 3

25 Feb 2026

·firstwordpharma.com

Biopharma bites: BioMarin's search for Roctavian buyer a bust, plus GSK's Frontier deal, and FDA updates from Immedica, MacroGenics

With no buyer, BioMarin pulls gene therapy from shelvesGSK inks $1B kidney-focused siRNA pactFDA approves Immedica's ultra-rare metabolic disease therapyPatient death triggers partial hold of MacroGenics' mid-stage cancer studyWith no buyer, BioMarin pulls gene therapy from shelvesHaving failed to find a buyer for its haemophilia A gene therapy, BioMarin Pharmaceutical said Monday that it will withdraw Roctavian (valoctocogene roxaparvovec-rvox) from the market. The company announced plans to divest the programme in October, after the therapy posted third-quarter sales of just $3 million. According to BioMarin, it "undertook a comprehensive effort to identify a potential buyer."In connection with its decision to pull Roctavian from shelves, BioMarin said it will take a $240 million charge, including a $119-million inventory write-off. -Elizabeth EatonGSK inks $1B kidney-focused siRNA pact GSK has agreed to shell out $40 million upfront for global rights to two siRNA programmes from China's Frontier Biotechnologies in a deal that could net the Chinese drugmaker up to $963 million in milestones, plus tiered royalties (see – Vital Signs: Charting China's course westward into 2026).In return, the UK drugmaker obtains exclusive worldwide rights to the two assets; one candidate is at the IND stage, while the second remains preclinical. Frontier will advance one programme through a Phase I study in China and complete IND-enabling work on the other before GSK assumes responsibility for global clinical development, regulatory filings and commercialisation."This agreement further strengthens our immunology pipeline, adding two potential first-in-class oligonucleotide therapies with significant opportunity to improve patient outcomes across multiple kidney diseases," said Kaivan Khavandi, global head for respiratory, immunology and inflammation R&D at GSK.The Frontier pact follows GSK's licensing deal with Empirico last October for rights to a Phase I siRNA programme, EMP-012, in chronic obstructive pulmonary disease. Empirico received $85 million upfront and is eligible for up to $660 million in milestones.-Anna BratulicFDA approves Immedica's ultra-rare metabolic disease therapyThe FDA on Monday awarded Immedica Pharma's arginine specific enzyme therapy, Loargys (pegzilarginase-nbln), with an accelerated approval to treat hyperargininaemia in patients 2 years and older with arginase 1 deficiency (ARG1-D) in conjunction with dietary protein restriction. The treatment is expected to be available in the US starting in April. The clearance was based on findings from the Phase III PEACE trial, in which Loargys significantly reduced plasma arginine from baseline compared with placebo at 24 weeks. According to Immedica, the therapy is the first and only treatment to address persistently elevated levels of plasma arginine, the disease's main driver. ARG1-D is an ultra-rare progressive metabolic disease that affects an estimated 250 people living in the US.-Elizabeth EatonPatient death triggers partial hold of MacroGenics' mid-stage cancer studyMacroGenics said Monday that the FDA has placed a partial clinical hold on the Phase II LINNET study of its experimental cancer drug lorigerlimab following a patient death, pausing patient enrolment. The company reported safety events that occurred across four patients, including two cases of grade 4 thrombocytopenia, one report of grade 4 myocarditis, and one patient with grade 4 neutropenia and concurrent septic shock, which resulted in their death. MacroGenics has dosed 41 patients with platinum-resistant ovarian cancer or clear cell gynaecologic cancer with lorigerlimab, a bispecific DART molecule that targets PD-1 and CTLA-4. Though enrolment is paused, study participants can continue to receive lorigerlimab."MacroGenics is fully committed to working closely with the FDA to resolve the partial clinical hold and we intend to resume study enrollment as soon as possible," said CEO Eric Risser.-Elizabeth Eaton

Phase 3Phase 2License out/inDrug ApprovalClinical Result

24 Feb 2026

·www.fiercepharma.com

Immedica overcomes prior FDA rebuke for ultrarare disease med to score new approval

The FDA has signed off on Immedica Pharma's ultrarare disease treatment Loargys four years after it refused to review a marketing application for the recombinant human enzyme from another drugmaker. Four years after the FDA issued its most heavy-handed form of a rejection to the prior company behind pegzilarginase, the U.S. regulator has now given the treatment a thumbs-up.Scoring the accelerated nod is Immedica Pharma for Loargys as a therapy for hyperargininemia in the ultrarare genetic disorder Arginase 1 deficiency (ARG1-D). The approval covers patients age 2 and older, with the therapy to be used in conjunction with a protein-restricted diet. Loargys, which is also known as pegzilarginase, is a recombinant human enzyme designed to lower levels of arginine in patients who are unable to break down the amino acid. It is the first treatment to address the elevated levels of plasma arginine associated with the disorder. The approval also comes after the FDA issued a complete response letter (CRL) to the company in August of last year citing major deficiencies across efficacy, dosing/administration, safety and labeling.ARG1-D affects an estimated 250 people in the U.S., with the current standard of care relying on symptom management including diet restrictions, arginine-free amino acid supplementation and nitrogen scavenging agents. Patients are often diagnosed in late infancy or early childhood, and the symptoms include spasticity, seizures and intellectual disability. “The accelerated approval of Loargys offers a fundamentally new approach that addresses the enzyme deficiency itself,” Stephen Cederbaum, M.D., a professor of human and medical genetics at the University of California, Los Angeles, said in a release. “Since persistently elevated arginine and its metabolites have been reported to be the proximal or direct driver of disease progression, this is a major advancement in metabolic medicine.” The company expects to make Loargys—which is infused or injected weekly—available in the U.S. in April. The green light comes after the FDA dismissed a marketing application for pegzilarginase in 2022 from Texas-based Aeglea BioTherapeutics, handing down a refusal-to-file (RTF) letter. An RTF is issued when the agency determines an application cannot be reviewed due to deficiencies that cannot be quickly resolved.A year after the rejection, Aeglea sold pegzilarginase to its European partner Immedica for $15 million upfront and $100 million in potential milestones.While a clinical trial of pegzilarginase linked it to an 80% reduction in mean plasma arginine compared to placebo, there were questions about whether it improved the health of patients. While mobility scores for patients improved in the treatment arm and worsened in the placebo group, the difference fell short of statistical significance. To ultimately gain FDA approval of Loargys, Immedica relied on results from the same trial, while also submitting additional "long-term data," it said in an email. "The application was resubmitted with additional data, analyses and clarifications addressing each of the FDA’s objections in the refusal to file and advice in regulatory interactions, which ultimately supported the agency’s decision to accept the filing for review," the company added.Additionally, in the August CRL, the FDA suggested the possibility of an accelerated-approval pathway "only with a postmarketing confirmatory trial designed to validate surrogate endpoints and demonstrate clinical benefit."In December 2023, Sweden-based Immedica earned marketing approval of Loargys from the European Medicines Agency. The private company, which was established in 2018 by Nordic investment firm Impilo, does not report sales of its products.At the end of 2024, Immedica acquired troubled Marinus Pharmaceuticals and its seizure medicine Ztalmy, which was approved in 2022.EDITOR'S NOTE: Immedica's responses to questions about how it ultimately gained FDA approval of Loargys were added to the story after it was initially published.

Drug ApprovalAccelerated Approval

100 Deals associated with Pegzilarginase

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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
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

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06582524 (CAEB1102-301A, Company_Website) Manual	Phase 3	Hyperargininemia	3	pegzilarginase	wxbixqeqmt(wzknykwhbz) = clinically meaningful reduction onzhhtfsob (xmaslvhoul ) View more	Positive	02 Feb 2026
NCT03921541 (PEACE, CTgov)	Phase 3	Hyperargininemia	32	Pegzilarginase (Pegzilarginase)	gtovwkeswj(fuxyvaojrx) = lqmcrznhcu wmpctbabxs (iuzrvlqqrd, 1.635) View more	-	19 Nov 2024
NCT03921541 (PEACE, CTgov)	Phase 3	Hyperargininemia	32	Placebo (Placebo)	gtovwkeswj(fuxyvaojrx) = yodsrogwrg wmpctbabxs (iuzrvlqqrd, 1.371) View more	-	19 Nov 2024
NCT03921541 (PEACE, SSIEM2022)	Phase 3	Hyperargininemia plasma arginine (pArg)	32	Pegzilarginase	tdottjukjc(utxpewvyhw) = xtfdqvefwy vixxwlsbpu (xpaodyxpdd )	Positive	30 Aug 2022
NCT03378531 \| NCT02488044 (Pubmed \| J Inherit Metab Dis) Manual	Phase 1/2	Hyperargininemia	16	pegzilarginase	qyolscwjul(ddwpejbtob) = five patients were all observed in 101A dqkncnzkdp (mbxkonpfkf ) View more	Positive	01 Jul 2021
SSIEM2019	Phase 1/2	Hyperargininemia plasma arginine	16	Pegzilarginase	dutssbkhaq(zreiwfjqot) = bbjvaohbew yvvwgeuygi (sjbdemyolq )	-	25 Aug 2019
SITC2018	Phase 1	Neoplasms	-	Pegzilarginase	avgszwiviy(kzyuxnxjjv) = increased levels of granzyme A, IFN-g and TNF-a ybrrgwunqu (tcoeyszcgd ) 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	lrqacnowis(rjcvdyuxmp) = 0.33 mg/kg once weekly dbzkcvngdg (pmakbkgikf ) View more	Positive	01 Jul 2018
NCT02732184 (ASCO 12018 \| ASCO 22018) Manual	Phase 1	Refractory acute myeloid leukemia \| Myelodysplastic Syndromes	21	pegzilarginase	kfgkipjocz(dtjlcfvfdw) = rinwryhlur ttcdzxmuwx (sigsxyxqma )	Positive	04 Jun 2018

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