Agalsidase beta biosimilars
An enzyme replacement for Fabry disease, with a single biosimilar in the world, approved in Japan in 2018.
About agalsidase beta
Agalsidase beta is a manufactured copy of an enzyme that people with Fabry disease lack. Without it, a fatty substance builds up in the kidneys, heart and nerves. The enzyme is infused to clear it.
The enzyme is taken up into cells through a sugar tag on its surface and breaks down the stored fat inside them. The sugar tag is what makes it work, and it is the hardest part to copy.
An infusion into a vein every two weeks, for life.
Fabry disease.
Fabry disease is very rare, so a biosimilar is compared mainly in the laboratory, especially its sugar tags, with a small clinical comparison in patients using a blood marker of the stored fat.
The reference product is Fabrazyme, from Sanofi.
Agalsidase beta is in the Atlas as the only enzyme replacement therapy with a biosimilar anywhere, approved in Japan by JCR in 2018. Japan has approved biosimilars that no other regulator has, and this one may be followed by other enzyme therapies.
Clinical details are summarised from the reference product's prescribing information. Approval dates, numbers and presentations are in the tables below, each with its source.
Competitive outlook
- Todayone agalsidase beta biosimilar is approved in at least one of the seven markets.
- AheadNo further candidate is visible in comparative trials or under review, so the competitive set is the approved products in the register below.
No published switching study for this molecule yet.
Approvals by year
Regulatory register
| Regulator | Brand | Holder or applicant | Approved | Application or permission | US licence | Status | Source |
|---|---|---|---|---|---|---|---|
| PMDA | Agalsidase Beta BS [JCR] | JCR Pharmaceuticals | Sep 2018 | not published | Approved | S020 |
Last checked Sep 2026. US interchangeability designations are listed in full on the interchangeable biosimilars page. CDSCO lists permissions by firm and INN, not brand. PMDA's list and the UAE directory do not publish application numbers; Canadian dates are the first approval or market date recorded in the Drug Product Database.
Regulatory pathway by market
Requirements for an agalsidase beta biosimilar under the guidance in force in September 2026.
| FDA, United States | EMA, European Union | MHRA, United Kingdom | PMDA, Japan | Health Canada | CDSCO, India | EDE, UAE | |
|---|---|---|---|---|---|---|---|
| Legal pathway | Section 351(k) of the Public Health Service Act (BPCIA 2009) | Article 10(4) of Directive 2001/83/EC; centralised procedure is mandatory for biotechnology products | UK marketing authorisation from MHRA. EU approvals in force at the end of 2020 became Great Britain licences automatically; new products since 2021 are approved through reliance on EMA or FDA decisions, and from 2024 through the International Recognition Procedure. Northern Ireland follows EU licences | MHLW approval on PMDA review under the Guideline for Ensuring Quality, Safety and Efficacy of Biosimilars (2009, revised 2020), with Q&A updates in 2024 and 2026 | Notice of Compliance under the Food and Drug Regulations as a biosimilar biologic drug, assessed under Health Canada's guidance on biosimilars (2016, revised 2022 and 2026) | Guidelines on Similar Biologics (CDSCO and DBT, 2016, in force); revised draft published May 2025 | Registration through the Emirates Drug Establishment; the public directory does not distinguish biosimilars from other biologicals |
| Reference product | US-licensed reference; a non-US comparator may be used with analytical and PK bridging | EU-authorised reference; a non-EEA comparator may be used with bridging data | Reference biologic authorised in the UK, or an EU-authorised version with bridging data | The original biopharmaceutical approved in Japan; foreign-sourced reference accepted with bridging since the 2020 revision | Reference biologic drug authorised in Canada, or a non-Canadian version of it with bridging data | Product licensed in India, or if not, one licensed in an ICH country, with justification | Not stated in the public directory; registration relies on assessment by reference regulators |
| Comparative clinical study | Draft guidance (October 2025): comparative analytical assessment plus a PK similarity study and immunogenicity assessment; a comparative efficacy study is generally not needed | Reflection paper adopted March 2026: a comparative efficacy trial can be waived when analytical and PK comparability is sufficient | Follows the EU guideline; MHRA's 2021 biosimilar guidance states that a comparative efficacy trial is generally not needed where analytical and pharmacokinetic comparability is shown | Comparative PK study and, in principle, a comparative efficacy study; the May 2026 early consideration sets out when the efficacy study can be omitted, and Japanese patient data are no longer required in every case (2025) | Comparative structural and functional studies, PK and, where warranted, PD equivalence; the 2026 revision reduces the expectation of comparative efficacy trials in line with FDA and EMA | Comparative PK/PD study and a comparative safety and efficacy study are generally expected, followed by a post-marketing study | Assessed on the dossier accepted by the reference regulator |
| Interchangeability | A separate designation permitting pharmacy-level substitution; switching studies generally not required under the June 2024 draft | EMA and HMA statement (September 2022): EU biosimilars are interchangeable with their reference; substitution rules are set by each member state | No designation; MHRA considers approved biosimilars interchangeable with their reference, and switching is decided by the prescriber and by NHS commissioning | Not defined; prescribing by brand, with pharmacist substitution not automatic | No federal designation; substitution and mandatory switching policies are set by each province, several of which have required switching since 2019 | Not defined in the guidelines | Not defined |
| Naming | INN plus a four-letter suffix | INN, identical to the reference; brand name and batch recorded for pharmacovigilance | Brand name with the non-proprietary name; licence numbers PL, PLGB or PLNI | INN followed by BS (biosimilar), strength, form and the company name in quotation marks, with a biosimilar number in the non-proprietary name | Non-proprietary name identical to the reference, with brand name and DIN as identifiers | INN with the brand name | Brand name as registered |
Sources: FDA draft guidance on comparative efficacy studies (October 2025) and interchangeability (June 2024); EMA reflection paper EMA/CHMP/BMWP/60916/2025 (March 2026) and EMA/HMA statement on interchangeability (September 2022); CDSCO Guidelines on Similar Biologics 2016 and 2025 draft (S009, S010); EDE directory (S018); MHLW/PMDA biosimilar guideline and notifications (S021); Health Canada guidance on biosimilar biologic drugs; MHRA guidance on the licensing of biosimilar products (2021).
EvySaif prepares the comparative evidence plan and the regional dossier strategy for agalsidase beta programmes. Regulatory strategy consulting
Presentations and concentration
5 mg and 35 mg lyophilised vials for infusion.
| Product | FDA | EMA | MHRA | PMDA | HC | CDSCO | EDE |
|---|---|---|---|---|---|---|---|
| Agalsidase Beta BS JCR | · | · | · | 5 mg and 35 mg vials | · | · | · |
EU presentations are listed in each product's SmPC.
Developers by market
| Developer | Product | FDA | EMA | MHRA | PMDA | HC | CDSCO | EDE |
|---|---|---|---|---|---|---|---|---|
| JCR Pharmaceuticals | Agalsidase Beta BS JCR | · | · | · | ✓ | · | · | · |
Entering a market where a competitor is already listed changes the evidence and pricing conversation. EvySaif's global value dossier and HTA submission work covers that side.
Products
In development
No comparative trial of an agalsidase beta biosimilar candidate against the reference product was found on ClinicalTrials.gov with the word biosimilar in the record. That does not mean nothing is in development: programmes registered only on national registries, or under a code without the word, are not captured. See the pipeline page for how the list is built.
Questions
Is there an agalsidase beta biosimilar in the United States or Europe?
No. The only biosimilar anywhere is JCR's product in Japan.
Is there an agalsidase beta biosimilar in India?
No. The molecule does not appear in the Indian permission lists.
Is there an agalsidase beta biosimilar in the UAE?
No. Fabrazyme is registered.
Why is this the only enzyme replacement biosimilar?
Enzyme replacement therapies depend on sugar tags for their effect, treat very rare diseases, and are protected more by manufacturing know-how than by patents, so few developers have tried.
How was the Japanese biosimilar tested?
Laboratory comparison including the sugar tags, a study of how the enzyme behaves in the body, and a small clinical comparison in Fabry patients.
Could the Japanese product be approved elsewhere?
It would need a separate application in each market, and none appears in EMA's list of applications under review.
Work with EvySaif on agalsidase beta
Agalsidase beta biosimilar landscape report
The register on this page as a delivered document: every approval, holder, presentation and pathway requirement across FDA, EMA, CDSCO and the Gulf, updated to the month, with the sources attached.
Request the landscape reportRegulatory gap assessment
For a developer deciding whether and where to file: a review of your analytical, PK and clinical package against what each regulator expects for agalsidase beta, with the gaps ranked and a filing sequence recommended.
About the gap assessmentSources
S020PMDA, list of approved biosimilar products in Japan (as of July 2026) Primary (regulator)S016FDA Purple Book, monthly data downloads January to August 2026 Primary (regulator)S017EMA, medicines data table (medicines-output-medicines-report_en.xlsx) Primary (regulator)S018UAE Emirates Drug Establishment, registered medical product directory Primary (regulator)S009CDSCO and DBT, Guidelines on Similar Biologics 2016 Primary (regulator)S010CDSCO, Draft Guidelines on Similar Biologics 2025 Primary (regulator)
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