RegulationPriority Review (China), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China), Conditional marketing approval (China)

Structure/Sequence

Sequence Code 4987

Source: *****

Clinical Trials associated with Agalsidase alfa

NCT05067868

/ RecruitingPhase 4

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa [r-DNA Origin]) in Indian Children and Adults With Fabry Disease

The main aim of this study is to learn more about the safety profile of Replagal.
Participants will receive Replagal every 2 weeks at the clinic for about 1 year.

Start Date01 Nov 2022

Sponsor / Collaborator

Shire Plc

[+1]

NCT04974749

/ CompletedPhase 3

An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease

The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.

Start Date01 May 2022

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

CTRI/2021/12/038690

/ Active, not recruitingPhase 4

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase alfa (r-DNA origin) (Replagalâ?¢) in Indian Children and Adults With Fabry Disease - NA

Start Date30 Dec 2021

Sponsor / Collaborator

Shire Biotech India Pvt Ltd.

100 Clinical Results associated with Agalsidase alfa

100 Translational Medicine associated with Agalsidase alfa

100 Patents (Medical) associated with Agalsidase alfa

218

Literatures (Medical) associated with Agalsidase alfa

01 Jun 2026·Molecular Genetics and Metabolism Reports

Prevalence and sociodemographic, clinical, and genetic characteristics of Fabry disease in north-central Chile, 2013–2023

Article

Author: Cortes Rivera, Basthian ; Royo Martínez, Sergio ; Ramírez-Santana, Muriel ; Molt, Fernando ; Castillo-Montes, Mauricio

Background:

Fabry disease (FD) is a rare genetic disorder linked to X chromosome, causing progressive multisystem involvement. Epidemiological data on FD are limited, both in Latin America and Chile. This study aimed to estimate the prevalence of FD and describe the sociodemographic and clinical characteristics of affected individuals in a north-central region of Chile.

Methods:

A descriptive cross-sectional study was conducted on 69 patients with FD during the period 2013-2023. An anonymized questionnaire collected data on sociodemographic variables, enzyme replacement therapy (ERT), chronic medications, clinical manifestations, and genetic variants. Descriptive statistics and correlation tests were used. Prevalence was estimated using official population data.

Results:

The regional prevalence rate was 8.44 per 100,000 inhabitants (95% CI: 6.44-10.43), with a particularly high rate in the commune of Coquimbo (22.45 per 100,000 inhabitants). The majority were women (58.0%), with a mean age of 35.8 years. 94% had the classical form of FD, and 98.6% carried the c.776C > G (p.P259R) variant. ERT was initiated in 2016 for 58% of patients, and 55.1% received agalsidase alfa. Peripheral nervous system involvement was present in 71.0%, and kidney involvement in 39.1%. The older the patient, the more systems are affected. 46.4% used complementary drugs, mostly analgesics, antihypertensives and antineuropathics, whose consumption increases with age.

Conclusions:

The Coquimbo Region has the highest prevalence of FD in Chile. Age at treatment initiation influences system involvement and chronic medication use. The results highlight the need for additional genetic, epidemiological, and clinical studies in the region.

01 Apr 2026·Journal of Allergy and Clinical Immunology-In Practice

Reply to “Fatal reactions to alpha-gal might be due to comorbid mast cell diseases”

Letter

Author: Richards, Nathan E ; Wilson, Jeffrey M ; McFeely, Erin ; Workman, Lisa J ; Platts-Mills, Thomas A E

01 Jan 2026·EUROPEAN JOURNAL OF CLINICAL INVESTIGATION

Long‐term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey

Article

Author: Schenk, Jörn ; Kampmann, Christoph ; Hughes, Derralynn A. ; Ramaswami, Uma ; West, Michael L. ; Reisin, Ricardo ; Nicholls, Kathleen ; Botha, Jaco ; Jain, Siddharth ; Anagnostopoulou, Christina ; Giugliani, Roberto ; Pintos‐Morell, Guillem ; Niu, Dau‐Ming

Abstract:

Background:

We present the final report from the Fabry Outcome Survey (FOS) on long‐term effectiveness and safety of agalsidase alfa in adults (≥18 years old).

Methods:

FOS was an international, multicentre, observational registry (NCT03289065), designed to enhance the understanding of Fabry disease and improve clinical management. Primary effectiveness endpoints were annualized change in estimated glomerular filtration rate (eGFR) and left ventricular mass index (LVMI), and time to and age at composite morbidity event (comprising renal, cardiac or stroke events) and death. Safety outcomes were also assessed.

Results:

FOS included data for 1864 adults (female/male, n = 907/957) who received agalsidase alfa only for a median (minimum, maximum) of 6.0 (0, 21.6) years, and 1613 untreated adults (female/male, n = 1235/378). At baseline, mean (standard deviation [SD]) eGFR was 94.01 (27.60) mL/min/1.73 m 2 in treated adults; annualized changes in eGFR (slope [standard error; SE]) remained relatively stable in females and declined slightly in males (−1.07 [.12] vs. −2.17 [.12] mL/min/1.73 m 2 ). At baseline, mean (SD) LVMI was 58.25 (25.01) g/m 2.7 and LVMI (slope [SE]) remained stable (.34 [.16] vs. .38 [.15] g/m 2.7 /year in females and males, respectively). Time (median [95% confidence interval]) from treatment initiation to first composite event was longer for females than males (83.4 [65.7–98.0] vs. 56.3 [45.6–66.7] months); age (median [minimum, maximum]) at death was also higher for treated females than males (69.9 [32.5, 87.7] vs. 59.1 [26.2, 79.6] years). Agalsidase alfa was generally well tolerated.

Conclusions:

This report further supports the long‐term effectiveness and safety of agalsidase alfa in adults with Fabry disease.

News (Medical) associated with Agalsidase alfa

28 Aug 2026

·www.biospace.com

Chiesi Global Rare Diseases Demonstrates Its Ongoing Commitment to the Rare Disease Community at the Society of Inborn Errors of Metabolism (SSIEM) 2026 Annual Symposium

-- Chiesi’s abstract presentations highlight clinical insights and patient-reported outcomes in Fabry disease and alpha-mannosidosis -- -- Rooted in patient voice and scientific rigor, this research reflects Chiesi’s long-standing role in advancing science for the lysosomal storage disorders community -- PARMA, Italy, Aug. 28, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced presentations at the Society of Inborn Errors of Metabolism (SSIEM) 2026 Annual Symposium, held August 25-28, 2026, in Helsinki, Finland. The company is proud to support 13 scientific abstracts across Fabry disease and alpha-mannosidosis, including 6 Chiesi-led presentations and 7 independent studies supported by Chiesi through scientific research grants and educational projects. These abstracts reflect the scope of the Company's scientific engagement with the lysosomal storage disorders (LSDs) and rare disease communities. These abstracts highlight the breadth of research advancing the understanding of Fabry disease and alpha-mannosidosis. Chiesi-led presentations include long-term Phase 3 data from the F60/BRILLIANCE trial, patient-reported outcomes from a cross-sectional survey in untreated Fabry disease, and research exploring earlier diagnosis and genotype–phenotype relationships in alpha-mannosidosis. Independent studies supported by Chiesi examine real-world clinical experience, disease biology, biomarkers and newborn screening. Together, this research contributes to a growing body of evidence to advance understanding of disease burden, diagnosis and long-term management in rare diseases. “Rare disease care demands patience, precision, and an unwavering focus on the people we're ultimately trying to help. The data presented at this year's SSIEM Annual Symposium reflects years of collaboration with investigators, clinicians, and the Fabry disease and alpha-mannosidosis communities,” said Enrico Piccinini, Senior Vice President, Europe and International, Chiesi Global Rare Diseases . “From new tools that could help identify diagnosis earlier to long-term data on treatment outcomes, this research is united by a single goal: better understanding the experiences of people living with rare diseases and using those insights to help inform care. We're proud to have shared these findings with the broader scientific community and remain devoted to advancing solutions that make a meaningful difference.” “This body of evidence spans long-term clinical trial data, real-world outcomes, and new biomarker research, contributing to a more comprehensive understanding of disease progression and unmet needs across the care journey,” said Alessio Amadasi, Vice President, Medical Affairs EU & International, Chiesi Global Rare Diseases . “That kind of understanding informs better care today while helping to shape the future of rare disease management.” Beyond the data presented at this year's Congress, Chiesi Global Rare Diseases recognizes that advancing care for people living with lysosomal storage disorders requires continued learning, collaboration, and investment in research. While the findings shared at SSIEM contribute to a growing body of knowledge, important questions remain across diagnosis, disease understanding, and long-term care. That is why Chiesi develops initiatives such as Find For Rare, now in its second edition, an independently assessed, expert-led research grant initiative designed to enable innovative research in lysosomal storage disorders. By providing funding opportunities through an independent selection process, Find For Rare helps advance knowledge in areas that matter most to the rare disease community and reflects Chiesi's long-term commitment to supporting progress for people living with these conditions today and for those who come next. Details of Chiesi-led presentations are as follows: Fabry disease Title: The reality of untreated Fabry disease: patient-reported challenges from a 238-participant cross-sectional survey Format : Poster Presentation (Poster #87) Presenter: Dawn Laney, M.S., Emory University School of Medicine Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Title: Long-term safety and efficacy of pegunigalsidase alfa in treatment-naive Fabry disease patients in F60/BRILLIANCE trial Format : Poster Presentation (Poster #539) Presenter: Ozlem Goker-Alpan, M.D., Lysosomal and Rare Disorders Research and Treatment Center Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Title: Long-term safety and efficacy of pegunigalsidase alfa in patients who switched from agalsidase alfa Format : Poster Presentation (#739) Presenter: Robert Hopkin, M.D., Cincinnati Children's Hospital Medical Center Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Title: Tolerability of 1-Hour Infusions of Pegunigalsidase Alfa in Fabry Disease: Data from the F60/BRILLIANCE Trial. Format : Poster Presentation (#529) Presenter: Robert Hopkin, M.D., Cincinnati Children's Hospital Medical Center Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Alpha-mannosidosis Title: Detection of alpha-mannosidosis: a phenotypic similarity approach powered by natural language processing Format : Poster Presentation (#284) Presenter: Nathalie Guffon, M.D., Femme Mère Enfant Hospital Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Title: Clinical impact of the MAN2B1 c.2248C>T variant in patients with alpha-mannosidosis: genotype–phenotype insights from the SPARKLE registry Format : Poster Presentation (#314) Presenter: Nicole Muschol, M.D., University Medical Center Hamburg-Eppendorf Date/Time: Wednesday, August 26, 5:30-7:30 pm EEST Details of independent presentations supported by Chiesi are as follows: Fabry disease Title: Pegunigalsidase alfa improves exercise tolerance and skeletal muscle bioenergetics in a Fabry disease mouse model Format: Poster Presentation Presenter: Jessica Gambardella, Ph.D. Federico II University of Naples, Naples, Italy Title: Real-world experience with the new four-week dosing regimen of pegunigalsidase alfa in Fabry disease: a multicenter study Format: Poster Presentation Presenter: Alicia Ugena, MD, Hospital Universitario de Cuenca, Cuenca, Spain Alpha-mannosidosis Title: Integrating α-mannosidosis into the differential diagnostic algorithm for suspected MPS: A 30-month update Format : Poster Presentation Presenter: Berthold Streubel, M.D., Medical University of Vienna Title: Alpha-Mannosidosis: Biomarker-based Treatment Monitoring Format : Poster Presentation Presenter: Dominik Dörfel, University Medical Center Hamburg-Eppendorf Title: Secondary Mitochondrial Dysfunction in Alpha-Mannosidosis: Implications for Disease Pathophysiology and Therapeutic Targeting Format : Oral Platform Presentation Presenter: Mollie Dewsbury, Liverpool John Moores University Title: Integrated Clinical, Biochemical, and Genetic Characterization of α-Mannosidosis in a Spanish Cohort: The α-REVEAL Study Format : Oral Platform Presentation Presenter: Belén Pérez, Ph.D., Autonomous University of Madrid (Center for the Diagnosis of Molecular Diseases) Title: Newborn screening for alpha-mannosidosis: quantitative assessment of a novel biomarker in dried blood spots Format : Oral Platform Presentation Presenter: Dominik Dörfel, University Medical Center Hamburg-Eppendorf Important Safety Information and Indication Elfabrio ® (pegunigalsidase alfa-iwxj) is a prescription infusion medicine used to treat adults with confirmed Fabry disease. Important Safety Information What is the most important information I should know about Elfabrio? Severe allergic reactions (hypersensitivity reactions), including anaphylaxis, may occur during and after Elfabrio treatment. If severe allergic reactions or anaphylaxis occurs during treatment, your healthcare provider will immediately stop the infusion and provide appropriate medical care. If these reactions should occur after treatment, seek immediate medical care. What should I know about Elfabrio infusions? Your healthcare provider may give you other medications prior to your Elfabrio infusions to help manage allergic reactions and infusion-related side effects. They will explain how to recognize the signs and symptoms of these allergic reactions and infusion-related side effects. If these signs and symptoms occur, it’s important for you to seek immediate medical care. If the reaction is mild to moderate, your healthcare provider may choose to slow the infusion rate or withhold the dose. In clinical trials, 41 patients (29%) experienced an infusion-related side effect. The most common signs and symptoms of an infusion-related reaction with Elfabrio were hypersensitivity, nausea, chills, itchy skin, rash, chest pain, dizziness, vomiting, feelings of weakness, pain, sneezing, shortness of breath, nasal congestion, throat irritation, abdominal pain, skin redness, diarrhea, burning sensation, nerve pain, headache, tingling or numbness, shaking movements, agitation, increased body temperature, flushing, slow heart rate, muscle pain, high blood pressure, and low blood pressure. Your healthcare provider will do blood and urine tests to check your kidney function during treatment with Elfabrio. The most common side effects of Elfabrio include infusion-related side effects, common cold, headache, diarrhea, fatigue, nausea, back pain, pain in the limbs, and sinus infection. Please see Full Prescribing Information for Elfabrio. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. Important Safety Information and Indication Lamzede ® (velmanase alfa-tycv) is intended for the treatment of non-central nervous system symptoms of alpha-mannosidosis in adult and pediatric patients. Important Safety Information What is the most important information I should know about Lamzede? Severe allergic reactions (hypersensitivity reactions) including anaphylaxis, may occur during and after Lamzede treatment. If severe allergic reactions or anaphylaxis occur during treatment, your healthcare provider will immediately stop the infusion and provide appropriate medical care. If these reactions should occur after treatment, seek immediate medical care. What should I know about infusions? Your healthcare provider may give you other medications prior to your next infusion to help manage allergic reactions and infusion-related side effects. They will explain how to recognize the signs and symptoms of these allergic reactions and infusion-related side effects. If these signs and symptoms occur, it’s important for you to seek immediate medical care. What are the common signs and symptoms of an allergic reaction or infusion-related side effects with Lamzede? In clinical trials, some patients experienced signs and symptoms of an allergic reaction, which included bluish skin discoloration, low blood pressure, vomiting, hives, skin redness, facial swelling, fever, and involuntary movements. In clinical trials, when patients experienced an infusion-related side effect, the most common signs were fever, chills, skin redness, vomiting, cough, itching, rash, and pink eye. Are there certain people who should or should not take Lamzede? Lamzede may cause harm to your unborn baby. For females who are able to become pregnant, your healthcare provider should do a pregnancy test before you start treatment with Lamzede. Tell your healthcare provider right away if you become pregnant or think you may be pregnant. You should use effective birth control during treatment with Lamzede and for at least 14 days after the last dose. What are the most common side effects of Lamzede? Lamzede can cause side effects including severe allergic reactions and anaphylaxis, common cold, fever, headache, and joint pain or stiffness. Please see Full Prescribing Information for Lamzede. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. About Fabry Disease Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body’s cells, affecting the heart, kidneys, skin, nervous system, and other organs. Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke. The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment — such as enzyme replacement therapy or pharmacological chaperone therapy — are critical in managing symptoms and slowing disease progression. About Alpha-mannosidosis Alpha-mannosidosis is an ultra-rare, inherited lysosomal storage disorder caused by mutations in the MAN2B1 gene, which results in a deficiency of the enzyme alpha-mannosidase. This deficiency leads to the accumulation of oligosaccharides, within the body’s cells, causing progressive damage to multiple organs and tissues. Alpha-mannosidosis can affect the musculoskeletal system, hearing, immune system, nervous system, and other organs, and is associated with a wide range of signs and symptoms, including skeletal abnormalities, impaired mobility, hearing loss, cognitive impairment, immune dysfunction, and behavioral or mental health challenges. The condition affects both children and adults and may present with few or mild symptoms early in life, progressing over time as oligosaccharides accumulate. While alpha-mannosidosis is rare, timely diagnosis and appropriate disease management are important to help address symptoms and support long-term outcomes for affected individuals. About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The Company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The Company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information visit . About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit . Follow @ChiesiGlobalRareDiseases on LinkedIn , Facebook , Instagram , X and YouTube . Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications Email: sstriar@lifescicomms.com PP-RA-02343 V1.0

17 Nov 2025

·www.prnewswire.com

First Recorded Case of Fatal Alpha-Gal Syndrome Highlights Uncommon Anaphylaxis Symptoms

This fatal case of delayed anaphylaxis raises concerns about lesser-known symptoms of food allergies and a growing prevalence of lone-star tick populations MILWAUKEE, Nov. 17, 2025 /PRNewswire/ -- A newly published case report in The Journal of Allergy and Clinical Immunology: In Practice (JACI: In Practice) details the first well-documented fatal case of alpha-gal syndrome (AGS) triggered by eating red meat. The report underscores several urgent clinical and public health concerns. Due to the limited exposure to AGS education, many Americans may be unaware that they are sensitized to alpha-gal, making the allergy difficult to diagnose and treat during an event of anaphylaxis. Additionally, AGS is commonly associated with delayed anaphylaxis, typically three to five hours after eating mammalian "red" meat, which makes the symptoms a challenge for patients to recognize. In the study, the patient experienced severe abdominal pain four hours after his consumption of beef products, "without any other allergic features" before his death. According to the researchers, severe delayed abdominal pain alone can be "a dangerous form of anaphylaxis [that] is not well recognized." The research suggests that an increase in deer population of many states between 1950 and 2020 is a cause for concern because "these deer are the primary breeding host of the Lone Star tick and are thought to be an important part of the reasons why this species of tick is moving north." The deceased patient was unaware that the "chigger" bites he experienced earlier that year could be Lone Star tick larvae, highlighting an important need for education and alpha-gal awareness as both patients and medical professionals in northern regions of the country may be unfamiliar with exposure to Lone Star tick populations. Because many clinicians in emerging tick regions may be unfamiliar with AGS, the authors stress the importance of awareness of delayed reactions, lone star tick migration and the need to consider AGS in cases of unexplained gastrointestinal distress or collapse. Read the full study. Visit aaaai.org to learn more about alpha-gal and red meat allergy. The American Academy of Allergy, Asthma & Immunology (AAAAI) is the leading membership organization of more than 7,100 allergists, asthma specialists, clinical immunologists and other professionals with a special interest in the research and treatment of allergic and immunologic diseases. Established in 1943, the AAAAI is the go-to resource for patients living with allergies, asthma and immune deficiency disorders. SOURCE American Academy of Allergy, Asthma & Immunology 21% more press release views with Request a Demo

Clinical StudyClinical ResultAACRImmunotherapy

04 Sep 2025

·investor.sangamo.com

Sangamo Therapeutics Presents Detailed Data from Registrational STAAR Study in Fabry Disease at International Congress of Inborn Errors of Metabolism 2025

Totality of data supports potential for isaralgagene civaparvovec as a one-time, durable treatment of the underlying pathology of Fabry disease to provide meaningful, multi-organ, clinical benefits above current standards of care STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as primary basis of approval Isaralgagene civaparvovec showed a favorable safety and tolerability profile Sangamo intends to submit a Biologics License Application (BLA) in 2026 under the Accelerated Approval pathway RICHMOND, Calif., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced detailed data from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. These data were presented at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in Kyoto, Japan, on September 4, 2025, in a poster presentation (Poster Ref: P-662). These data are also available on Sangamo’s website on the Presentations page. “These STAAR study data demonstrate the potential for ST-920 to provide meaningful clinical benefits to Fabry disease patients,” said Dr John Bernat, M.D., Ph.D., University of Iowa and investigator of the Phase 1/2 STAAR study. “The positive mean eGFR slope at both one and two years, which compares favorably to approved Fabry treatments, alongside stable cardiac function, are exciting developments, particularly given the decline in renal and cardiac function traditionally seen with Fabry patients. The ability for patients to discontinue the use of burdensome enzyme replacement therapies further supports the potential of ST-920 as a single-dose, durable treatment option for people living with Fabry disease.” “Fabry disease is a debilitating and multifaceted condition, for which there is a serious unmet medical need,” said Nathalie Dubois-Stringfellow, Ph. D, Chief Development Officer at Sangamo. “We are excited by the potential of ST-920 to provide long-lasting clinical benefits to a wide range of Fabry disease patients. Improvements in renal function and stable cardiac function, alongside quality of life and additional clinical benefits, show the ability of ST-920 to address the underlying pathology of Fabry disease and provide a potentially transformative treatment for Fabry disease patients. We look forward to sharing these data with health authorities.” Updated Phase 1/2 STAAR Study Results (as of April 10, 2025 cut-off date) Efficacy (32 dosed patients followed at least 12 months) A positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year (95% confidence interval (CI): -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients. This compares favorably to a meta-analysis of publications of approved Fabry treatments (Fabrazyme, Replagal and Galafold). Furthermore, a mean annualized eGFR slope at Week 104 of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who have achieved 104-weeks of follow-up. Supportive mean annualized eGFR slopes were also observed across a variety of patient subgroups, including gender, baseline ERT status, Fabry disease type and baseline eGFR, showing consistency in effect across Fabry patients in the study. Stable cardiac function was observed, including left ventricular mass (LVM), left ventricular mass index (LVMI), left ventricular myocardial global longitudinal strain (GLS), T1 and T2 mapping, end-diastolic and end-systolic volumes that remained stable over at least one year. Durability of effect was demonstrated with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for up to 4.5 years for the longest treated patient. All 18 patients who began the study on Enzyme Replacement Therapy (ERT) had been withdrawn from ERT and remained off ERT as of the data cutoff date1. Plasma lyso-Gb3 levels in these patients remained generally stable following ERT withdrawal. Of the 10 patients who had measurable titers of total antibodies (TAb) or neutralizing antibodies (Nab) against α-Gal A associated with ERT at baseline, TAb or NAb titers decreased markedly in nine patients and became undetectable in eight following treatment. Improvements in disease severity were reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI) age-adjusted score, with 22 patients showing improvements in their total MSSI score at 12 months and nine patients improving their FOS-MSSI disease category at the last assessment. Statistically and clinically significant improvements in the short form-36 (SF-36) quality of life scores were observed including role-physical +14.8 (95% CI: 7.3, 22.4, p=0.0003), vitality +9.6 (95% CI: 3.9, 15.2, p=0.0017), bodily pain +9.0 (95% CI: 2.3, 15.7, p=0.0104), social functioning +7.8 (95% CI: 2.0, 13.6, p=0.0100), general health +7.4 (95% CI: 2.0, 12.8, p=0.0091), and physical component scores +4.2 (95% CI: 1.8, 6.6, p=0.0014), at week 52 compared to baseline. Statistically significant improvements were seen in the gastrointestinal symptom rating scale (GSRS) compared to baseline. Safety (all dosed patients): Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning. The majority of adverse events were grade 1-2 in nature. The most common treatment-emergent adverse events (TEAEs) were pyrexia (60.6% of participants), COVID-19 (36.4%), headache (30.3%), and nasopharyngitis (33.3%). All TEAEs resolved in response to clinical management and there were no safety-related study discontinuations and no deaths. Sangamo believes these data support the potential for isaralgagene civaparvovec as a one-time, durable treatment of the underlying pathology of Fabry disease, to provide meaningful clinical benefits above current standards of care and will form the basis for an anticipated BLA submission under the Accelerated Approval pathway as early as the first quarter of 2026. The STAAR study enrolled male and female patients who were either on ERT, were ERT pseudo-naïve (defined as having been off ERT for six or more months), or were ERT-naïve. The median age of patients enrolled in the study was 42, with a median duration of follow-up of 24 months and the longest treated patient having achieved 4.5 years of follow-up. Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency. Sangamo is advancing BLA preparation activities for isaralgagene civaparvovec, while continuing to engage in business development negotiations for a potential Fabry commercialization agreement. A Current Report on Form 8-K summarizing the Phase 1/2 STAAR study data will be filed by Sangamo, and this press release is subject to the further detail provided in that Form 8-K. About the STAAR Study The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. About Fabry Disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities. About Sangamo Therapeutics Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders. Moreover, Sangamo’s SIFTER capsid discovery platform is advancing delivery to the central nervous system in preclinical studies. Sangamo is also progressing next generation genome editing through its modular integrase (MINT) platform. Sangamo’s pipeline includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter/X. 1 Since the data cutoff date, a physician has decided to resume ERT for one of their treated patients who had withdrawn from ERT. This patient, who received ST-920 more than two and a half years ago, maintained supraphysiological levels of a-Gal A activity, and their lyso-Gb3 levels were generally stable as of the data cutoff date. Forward-Looking Statements This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic and commercial potential of isaralgagene civaparvovec; the potential for isaralgagene civaparvovec to qualify for the FDA’s Accelerated Approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the potential BLA submission for isaralgagene civaparvovec, and the timing of such submission; Sangamo’s plans to seek a commercialization partner for ST-920; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo’s lack of capital resources and need for substantial additional funding to execute its operating plan and to continue to operate as a going concern, including the risk that Sangamo will be unable to obtain substantial additional funding on acceptable terms or at all or collaboration partners necessary to advance its preclinical and clinical programs, in particular for its Fabry disease program and to otherwise operate as a going concern, in which case Sangamo may be required to cease operations entirely, liquidate all or a portion of its assets and/or seek protection under the U.S. Bankruptcy Code; the uncertain timing and unpredictable nature of clinical trial results, including the risk that the therapeutic effects observed in the latest clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, including that the 104-week data from such study will not verify the clinical benefit of isaralgagene civaparvovec or support FDA approval, and that the patients withdrawn from ERT will remain off ERT; the effects of macroeconomic factors or financial challenges, including as a result of the ongoing overseas conflicts, tariffs, geopolitical instability, inflation and fluctuations in interest rates, on the global business environment, healthcare systems and business and operations of Sangamo and its collaborators; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results, including the results of any registrational trial of Sangamo’s product candidates; the potential for technological developments that obviate technologies used by Sangamo; Sangamo’s reliance on collaborators and Sangamo’s potential inability to secure additional collaborations; Sangamo’s ability to achieve expected future financial performance. All forward-looking statements about Sangamo’s future plans and expectations, including Sangamo’s development plans for its product candidates, are subject to Sangamo’s ability to secure adequate additional funding. There can be no assurance that Sangamo and its current or potential future partners will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2024, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law. Contacts Investor Relations Louise Wilkie ir@sangamo.com Media Inquiries Melinda Hutcheon media@sangamo.com Source: Sangamo Therapeutics, Inc.

Clinical ResultOrphan DrugFast TrackClinical Study

100 Deals associated with Agalsidase alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D02784	Agalsidase alfa	A16AB03	DB15874

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Fabry Disease	European Union	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	Iceland	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	Liechtenstein	Takeda Pharmaceuticals International AG	03 Aug 2001
Fabry Disease	Norway	Takeda Pharmaceuticals International AG	03 Aug 2001

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04974749 (CTgov)	Phase 3	Fabry Disease	20	REPLAGAL	enjtpgaygb = pnvixyzlor qiktxnravh (zwolzughfw, cbufvhvhfb - nuqhthjqtw) View more	-	08 Oct 2024
NCT04840667 (CTgov)	Phase 3	Fabry Disease	17	REPLAGAL	pqdhvkljrv(hgfyeazicz) = bxwzwbelpb kuqpkvralr (otrzmyluyy, uxgspiucwm - amtwuhkkxy) View more	-	17 Jun 2024
NCT01298141 (Pubmed \| Drugs R D) Manual	Phase 3	Fabry Disease	167	Agalsidase alfa	glomomkkui(btrssdztbf) = lgwhzngyac lhtgadleli (xboeskragk ) View more	Positive	01 Dec 2021
SSIEM2019	Not Applicable	Fabry Disease	493	Agalsidase alfa	hibgryveys(avqwspuodt) = pethegfllz otrlfdmabw (fahjpdvndc ) View more	Positive	25 Aug 2019
NCT01298141 (CTgov)	Phase 3	Fabry Disease	171	Replagal (Agalsidase Alfa)	liaoggnrkh = wzftlsownb daqwmtuaty (znsfixarpm, inhzfbudzp - btguuwvmtt) View more	-	15 Feb 2019
NCT01363492 (HGT-REP-084, Pubmed \| Drug Des Devel Ther) Manual	Phase 2	Fabry Disease	14	agalsidase alfa	ktobrlyqju(zhkbsiikol) = egrddfbfnj ghfqosblav (rpunuyskjn ) View more	Positive	25 May 2016
NCT01124643 (CTgov)	Phase 3	Fabry Disease	35	Replagal	pjyodarirc(dpeqyiedca) = fzakrdugcf najxbvmykb (beqijofriv, 13.46) View more	-	22 Aug 2014
NCT01363492 (HGT-REP-084, CTgov)	Phase 2	Fabry Disease	15	Replagal (Replagal (0.2 mg/kg))	dqeuzkhyem = nfqkwsymth xvzdcztdww (injfqfgsyc, wuhhonapbm - btpgshcvpk) View more	-	20 May 2014
NCT01363492 (HGT-REP-084, CTgov)	Phase 2	Fabry Disease	15	Replagal (Replagal 0.2 mg/kg)	olkyxiygve(rhqzwndvqy) = hsvxmryzey cueiwbemky (leyalszray, 24.223) View more	-	20 May 2014
NCT01304277 (CTgov)	Phase 2	Fabry Disease	17	agalsidase alfa	jczpqmvobm(wadrbrzmcg) = wtunclvwry dzjtfqirri (zcescvhsbz, vingdtsdwb - yjzvaoycfz) View more	-	25 Apr 2014
NCT00084084 (CTgov)	Phase 2	Fabry Disease	17	Agalsidase alfa	rzcteqqfyv = dsicufwbzo irgfaylrhi (qkedefwcef, nebfuiwldy - kolgxbaqmy) View more	-	10 Oct 2013

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