Do I need a prescription for Pegunigalsidase alfa 5mg/2.5ml solution for infusion vials?

Pegunigalsidase alfa 5mg/2.5ml solution for infusion vials is classified as a Valid as a prescribable product in the UK. However, always consult a pharmacist or doctor before use. (Source: NHS dm+d)

What are the brand names for Pegunigalsidase alfa 5mg/2.5ml solution for infusion vials?

Pegunigalsidase alfa 5mg/2.5ml solution for infusion vials is the generic name. It is available under brand names including: Elfabrio 5mg/2.5ml concentrate for solution for infusion vials. (Source: NHS dm+d — Actual Medicinal Products)

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Pegunigalsidase alfa 5mg/2.5ml solution for infusion vials

Prescription only

Requires a prescription from a doctor or prescriber

Infusion

5mg/2.5ml

Other

Oral liquids

Active ingredients:

Pegunigalsidase alfa

ATC classificationBrowse ATC index

International classification by the WHO, grouping medicines by the organ system they act on

ATC A16AB20

Chemical classBrowse classes

Classification based on the molecule's chemical structure and properties

Check interactions for Pegunigalsidase alfa

No active safety alerts

Official documents, adverse reaction reporting, and safety monitoring

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Official medicine documents

Yellow Card

Report side effects (MHRA)

Drug safety updates

MHRA alerts for Pegunigalsidase alfa

Source: MHRA Products DatabaseOGL 3.0

Updated 16 Jan 2026

Safety monitoring data

Yellow Card reports

MHRA

The MHRA Yellow Card scheme collects reports of suspected side effects from healthcare professionals and patients. View the Drug Analysis Profile (iDAP) for real-world adverse reaction data.

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Suspected adverse reactions reported for Pegunigalsidase alfa

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Submit a Yellow Card report to the MHRA

Data from the MHRA Yellow Card scheme. A reported reaction does not necessarily mean the medicine caused it. Contains public sector information licensed under the Open Government Licence v3.0.

EudraVigilance

EMA

The European Medicines Agency (EMA) collects suspected adverse reaction reports from across the EU/EEA through the EudraVigilance system. Search for safety data on this medicine.

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Suspected adverse reactions reported for Pegunigalsidase alfa

About EudraVigilance

Learn about EU pharmacovigilance and safety monitoring

EudraVigilance data is published by the European Medicines Agency (EMA). A suspected adverse reaction is not necessarily caused by the medicine.

1 branded products available

1 products

Possibly available on the UK marketDiscontinuedAvailability per NHS dm+d

MHRA licensed products

View all licensed products for Pegunigalsidase alfa on the MHRA register

Elfabrio 5mg/2.5ml concentrate for solution for infusion vials

Chiesi Ltd

Source: NHS dm+dOGL 3.0

Updated 1 Mar 2026

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Similarity is based on WHO Anatomical Therapeutic Chemical (ATC) classification and on a factual NHS dm+d therapeutic-grouping code prefix. Source data: NHS dm+d via TRUD (OGL v3.0), WHO ATC/DDD Index.

NHS prescribing volume and spending trends

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Guidelines from the National Institute for Health and Care Excellence

NICE clinical guidance(1)

Pegunigalsidase alfa for treating Fabry disease (TA915)

TA915

Technology appraisal

Updated Oct 2023

Source: National Institute for Health and Care Excellence (NICE). Contains public sector information licensed under the Open Government Licence v3.0.

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Supply & safety information

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UK regulator alerts and supply notices

Pharmacy links redirect to the retailer's own search and do not represent real-time stock levels. Shortage and safety information sourced from MHRA drug safety updates (gov.uk, Crown Copyright under OGL v3.0).

Codes for healthcare professionals and prescribing systems

These codes are used by healthcare IT systems and prescribers to identify this medicine.

NHS UK identifiers

SNOMED CT

44028311000001102

dm+d ID

44028311000001102

VTM (Virtual Therapeutic Moiety)

42389511000001102

VMP (Virtual Medicinal Product)

44028311000001102

International identifiers

ATC code — Anatomical Therapeutic Chemical (WHO)

A16AB20

Browse tools

dm+d Browser

NHSBSA medicines dictionary lookup

SNOMED CT and dm+d codes from NHS TRUD (Technology Reference data Update Distribution), licensed under the Open Government Licence v3.0. ATC codes from the WHO Collaborating Centre for Drug Statistics Methodology (whocc.no).

Active and completed clinical studies from ClinicalTrials.gov

Searching UK clinical trials...

Source: ClinicalTrials.gov, a database of the U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Data accessed via ClinicalTrials.gov API v2. Trial information is provided for research purposes and does not constitute medical advice.

Academic studies and reviews for this medicine's active substance

Showing the 50 most relevant studies.

Reviews & meta-analyses: 8 · Randomised trials: 21 · 1998–2026

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Interferon Alfa-2b Adjuvant Therapy of High-Risk Resected Cutaneous Melanoma: The Eastern Cooperative Oncology Group Trial EST 1684

J. M. Kirkwood, M. H. Strawderman, M. Ernstoff, et al.

Journal of Clinical Oncology, 2023

Randomised trial

Cited by 875

Randomized Phase III Trial of Pegvorhyaluronidase Alfa With Nab-Paclitaxel Plus Gemcitabine for Patients With Hyaluronan-High Metastatic Pancreatic Adenocarcinoma

E. Van Cutsem, M. Tempero, D. Sigal, et al.

Journal of Clinical Oncology, 2020

Randomised trial

Cited by 324

Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study.

H. Gisslinger, C. Klade, P. Georgiev, et al.

The Lancet. Haematology, 2020

Randomised trial

Cited by 310

Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial.

U. Platzbecker, M. D. Della Porta, V. Santini, et al.

Lancet, 2023

Randomised trial

Cited by 103

The Arecibo Legacy Fast ALFA Survey: The ALFALFA Extragalactic H i Source Catalog

M. Haynes, R. Giovanelli, B. Kent, et al.

The Astrophysical Journal, 2018

Review

Cited by 329

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.

J. Díaz-Manera, P. Kishnani, H. Kushlaf, et al.

The Lancet. Neurology, 2021

Randomised trial

Cited by 127

Bintrafusp alfa versus pembrolizumab in patients with treatment-naive, PD-L1-high advanced non-small cell lung cancer: a randomized, open-label, phase 3 trial.

B. Cho, J. Lee, Yi-long Wu, et al.

Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer, 2023

Randomised trial

Cited by 64

Ropeginterferon alfa-2b versus phlebotomy in low-risk patients with polycythaemia vera (Low-PV study): a multicentre, randomised phase 2 trial.

T. Barbui, A. Vannucchi, V. De Stefano, et al.

The Lancet. Haematology, 2021

Randomised trial

Cited by 102

Long-term outcomes of polycythemia vera patients treated with ropeginterferon Alfa-2b

J. Kiladjian, C. Klade, P. Georgiev, et al.

Leukemia, 2022

Randomised trial

Cited by 100

Efficacy and Safety of Daprodustat Compared with Darbepoetin Alfa in Japanese Hemodialysis Patients with Anemia: A Randomized, Double-Blind, Phase 3 Trial.

T. Akizawa, M. Nangaku, Taeko Yonekawa, et al.

Clinical journal of the American Society of Nephrology : CJASN, 2020

Randomised trial

Cited by 94

Sources: aggregated from Europe PMC (EMBL-EBI), OpenAlex, Crossref, PubMed and other open scholarly databases. Retracted articles are excluded. Study information is provided for research purposes and does not constitute medical advice.

Pharmacology and chemical data from DrugBank

go.drugbank.com

Key facts

Drug status

Approved

Major interactions

None known

Half-life

53 to 134 hours

Mechanism

Fabry disease is an X-linked recessive disorder caused by mutations in the GLA g…

Food interactions

None known

Human targets

2 targets

Data: DrugBank · CC BY-NC 4.0

Pharmacokinetics at a glance

Absorption

2 mg/k

The pharmacokinetic profile of pegunigalsidase alfa at 0.2, 1, and 2 mg/kg administered every two weeks was evaluated in adult patients with Fabry disease.…

Half-life

53 to 134 hours

Pegunigalsidase alfa given in two-week intervals has a plasma half-life that ranges from 53 to 134 hours across dose groups and visit day.
[L46342]

Protein binding

Not available.

Volume of distribution

1 mg/k

In enzyme replacement therapy-naïve patients with Fabry disease given an intravenous infusion of 1 mg/kg of pegunigalsidase alfa every two weeks,…

Metabolism

As a recombinant protein, pegunigalsidase alfa is expected to be metabolized through peptide hydrolysis.…

Elimination

Pegunigalsidase alfa has a molecular weight of approximately 116 kDa, which is twice the cut-off value for glomerular filtration.…

Clearance

2 mg/k

Patients treated with 1 and 2 mg/kg of pegunigalsidase alfa every two weeks had a higher half-life and AUC with increasing duration of treatment,…

Pharmacokinetic data: DrugBank · CC BY-NC 4.0

Status:

Approved

Investigational

Biologic

About this compound

Pegunigalsidase alfa (PRX-102) is a recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease, a rare genetic disorder characterized by the deficiency of alpha-galactosidase A.[A259352][L46342] Unlike other forms of recombinant alpha-galactosidase A, such as [agalsidase alfa] and [agalsidase beta], pegunigalsidase alfa uses a plant cell-based protein expression system, leading to a different glycosylation pattern. While agalsidase alfa and agalsidase beta enter lysosomes via the mannose-6-phosphate (M6P) receptor, pegunigalsidase alfa carries no M6P on its glycans and does not depend on the M6P receptor during cellular uptake. Furthermore, the pegylation of pegunigalsidase alfa promotes higher stability and a longer half-life, allowing it to reach target organs with a lower dose and frequency of administration.[A259347][A259352]

In May 2023, the EMA granted marketing authorization to pegunigalsidase alfa in the European Union (EU) for the treatment of adult patients with Fabry disease.[L46342][L46387] Later on, the FDA approved pegunigalsidase alfa for the treatment of adult patients with Fabry disease that same month.[L46432][L46437]

Properties:

liquid

DrugBank indication

Pegunigalsidase alfa is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).
[L46342][L46432]

Also known as(6)

Pegunigalsidase alfa

3.2.1.22

Alpha-D-galactosidase A

Alpha-D-galactoside galactohydrolase

Galactosylgalactosylglucosylceramidase GLA

Melibiase

Toxicity & overdose

During clinical studies, no cases of pegunigalsidase alfa overdose were reported. No specific signs and symptoms were identified with the maximum dose of pegunigalsidase alfa used (2 mg/kg body weight every two weeks). Infusion-related reactions and pain in extremities were the most commonly reported adverse reactions.

If an overdose is suspected, seek emergency medical attention.
[L46342]

How it works

Mechanism of action

Fabry disease is an X-linked recessive disorder caused by mutations in the GLA gene that lead to the loss of function of α-galactosidase A. This α-galactosidase A deficiency leads to the accumulation of globotriaosylceramide (GB3) and other neutral glycosphingolipids throughout the body, including the heart, kidney, nervous system, vascular endothelium and smooth muscle. This accumulation of GB3 is characterized by chronic pain, ocular opacities, liver and kidney impairment, skin lesions, vascular deterioration and cardiac deficiencies.[A259347][A259352][A259357]

Pegunigalsidase alfa is a pegylated recombinant form of human α-galactosidase-A, with an amino acid sequence similar to the human enzyme that replaces α-galactosidase-A. It promotes the hydrolysis of the terminal α-galactosyl moieties of oligosaccharides and polysaccharides in the lysosome, reducing the amount of GB3 accumulated in patients with Fabry disease.[L46342] The deacylated form of GB3, globotriaosylsphingosine (lyso-GB3), also plays a role in the development of Fabry disease, and the use of pegunigalsidase alfa decreases the accumulation of this metabolite as well.[A259352][L46342]

Pharmacodynamics

In patients with Fabry disease, the use of pegunigalsidase alfa led to a mean decrease in peritubular capillary globotriaosylceramide (GB3) content of 67.8% ± 8.9% after 6 months. At the 6-months time point, most patients had their GB3 levels reduced by more than half.[A259367] The use of pegunigalsidase alfa is associated with infusion-related reactions, hypersensitivity reactions and immunogenicity. The use of enzyme replacement therapies such as pegunigalsidase alfa may lead to depositions of immune complexes as a manifestation of immunological response. During the clinical development of pegunigalsidase alfa, a single case of glomerulonephritis membranoproliferative was reported. This led to a temporary decline in renal function, which improved upon the discontinuation of pegunigalsidase alfa.[L46342]

Pharmacokinetics

How the body processes this drug — absorption, distribution, metabolism, and elimination

Absorption

The pharmacokinetic profile of pegunigalsidase alfa at 0.2, 1, and 2 mg/kg administered every two weeks was evaluated in adult patients with Fabry disease. At these dose levels, the AUC_0-∞ of pegunigalsidase alfa increased with increasing doses on day one and throughout the study. Also, the mean values for dose-normalized AUC_0-2wk were for all doses, suggesting that pegunigalsidase alfa follows a linear dose-proportional pharmacokinetic profile.
[L46342]

In enzyme replacement therapy-naïve patients with Fabry disease given an intravenous infusion of 1 mg/kg of pegunigalsidase alfa every two weeks, the C_max went from 11.1 μg/mL on day 1 to 17.3 μg/mL on week 52, and the AUC_inf went from 391 μg·h/mL on day 1 to 1428 μg·h/mL on week 52.

In enzyme replacement therapy-experienced patients with Fabry disease given the same dose, the C_max ranged from 21.2 to 23.3 μg/mL, and the AUC_tau ranged from 958 to 1074 μg·h/mL.
[L46432]

Half-life

Pegunigalsidase alfa given in two-week intervals has a plasma half-life that ranges from 53 to 134 hours across dose groups and visit day.
[L46342]

Protein binding

Not available.

Volume of distribution

In enzyme replacement therapy-naïve patients with Fabry disease given an intravenous infusion of 1 mg/kg of pegunigalsidase alfa every two weeks, the volume of distribution was 321 mL/kg on day 1, 271 mL/kg on week 13, 226 mL/kg on week 26, and 186 mL/kg on week 52.
[L46432]

Metabolism

As a recombinant protein, pegunigalsidase alfa is expected to be metabolized through peptide hydrolysis. Therefore, impaired liver function is not expected to have a clinically significant effect on the pharmacokinetics of pegunigalsidase alfa. Based on its metabolism, pegunigalsidase alfa is an unlikely target of cytochrome P450 enzymes.
[L46342]

Route of elimination

Pegunigalsidase alfa has a molecular weight of approximately 116 kDa, which is twice the cut-off value for glomerular filtration. Because of this, filtration and/or proteolytic degradation in kidneys is unlikely.
[L46342]

Clearance

Patients treated with 1 and 2 mg/kg of pegunigalsidase alfa every two weeks had a higher half-life and AUC with increasing duration of treatment, as well as lower clearance and elimination volume of distribution, suggesting a saturated clearance.
[L46342]

In enzyme replacement therapy-naïve patients with Fabry disease given an intravenous infusion of 1 mg/kg of pegunigalsidase alfa every two weeks, clearance was 2.9 mL/h/kg on day 1, 2.3 mL/h/kg on week 13, 1.6 mL/h/kg on week 26, and 1.1 mL/h/kg on week 52.
[L46432]

Drug targets(2)

Proteins and enzymes this drug interacts with in the body

Globotriaosylceramide

BE0004830

metabolizer

ligand

Alpha-galactosidase A

BE0002422

GLA

modulator

Specific functionalpha-galactosidase activity

Cellular location

Lysosome

General function & pharmacology

Catalyzes the hydrolysis of glycosphingolipids and participates in their degradation in the lysosome

Scientific references(4)

Kizhner T., Azulay Y., Hainrichson M., Tekoah Y., Arvatz G., Shulman A., Ruderfer I., Aviezer D., Shaaltiel Y.

Characterization of a chemically modified plant cell culture expressed human alpha-Galactosidase-A enzyme for treatment of Fabry disease.

Molecular Genetics Metabolism

2015 Feb114(2):259-67. doi: 10.1016/j.ymgme.2014.08.002. Epub 2014 Aug 10

PMID: 25155442 DOI: 10.1016/j.ymgme.2014.08.002

Ruderfer I., Shulman A., Kizhner T., Azulay Y., Nataf Y., Tekoah Y., Shaaltiel Y.

Development and Analytical Characterization of Pegunigalsidase Alfa, a Chemically Cross-Linked Plant Recombinant Human alpha-Galactosidase-A for Treatment of Fabry Disease.

Bioconjug Chemistry

2018 May 1629(5):1630-1639. doi: 10.1021/acs.bioconjchem.8b00133. Epub 2018 May 3

PMID: 29698600 DOI: 10.1021/acs.bioconjchem.8b00133

Azevedo O., Gago M.F., Miltenberger-Miltenyi G., Sousa N., Cunha D.

Fabry Disease Therapy: State-of-the-Art and Current Challenges.

International Journal Molecular Science

2020 Dec 2822(1):206. doi: 10.3390/ijms22010206

PMID: 33379210 DOI: 10.3390/ijms22010206

Schiffmann R., Goker-Alpan O., Holida M., Giraldo P., Barisoni L., Colvin R.B., Jennette C.J., Maegawa G., Boyadjiev S.A., Gonzalez D., Nicholls K., Tuffaha A., Atta M.G., Rup B., Charney M.R., Paz A., Szlaifer M., Alon S., Brill-Almon E., Chertkoff R., Hughes D.

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.

Journal Inherit Metabolism Diseases

2019 May42(3):534-544. doi: 10.1002/jimd.12080. Epub 2019 Apr 8

PMID: 30834538 DOI: 10.1002/jimd.12080

ATC classification(1 code)

ATC A16AB20

Affected organisms(1)

Humans and other mammals

Experimental properties(2)

Chemical identifiers

CAS, UNII, InChI Key and database cross-references

Show

Linked compound data from DrugBank Open Data (CC BY-NC 4.0)