New data show the therapy led to minimal changes in renal function, even after more than 8 years.
New long-term data show that patients with Fabry disease who are treated with migalastat (Galafold) experienced stable renal function after 2 years or more on the drug.
The report offers some of the first data regarding the long-term data regarding the medication, which was approved by the FDA in 2018. The study was published in the journal Molecular Genetics and Metabolism Reports.
Fabry disease is a rare lysosomal storage disease caused by a mutation of the GLA gene. The mutation leads to buildup of globotriaosylceramide (Gb3) throughout the body, including in the kidneys. Renal manifestations of the diseaseas well as likely associated cardiac eventsare a significant cause of mortality and morbidity in patients with Fabry, noted corresponding author Daniel G. Bichet, MD of the University of Montreal, and colleagues.
Enzyme replacement therapy (ERT), in combination with the oral chaperone migalastat, has become the standard treatment for people with Fabry disease. The investigators explained that migalastat works by targeting the lysosomal enzyme lysosomal enzyme -galactosidase A (-Gal A), which is functionally deficient in patients with Fabry.
As a small molecule pharmacological chaperone, migalastat binds to and stabilizes amenable mutant forms of -Gal A in the endoplasmic reticulum, facilitating trafficking of -Gal A to lysosomes and restoring endogenous enzyme activity, Bichet and colleagues wrote.
Previous reports, including the phase 3 clinical trials for migalastat, showed that patients on the medication had stable renal function. However, those studies were based on shorter time frames of 24 months or less.
In the new study, the authors conducted a post-hoc analysis to look at the long-term renal-function changes in patients with amenable GLA variants who were given migalastat for 2 or more years as part of the phase 3 trials and/or long-term open-label extension studies.
A total of 78 patients were included in the analysis. Of those, 36 were ERT-naive, and 42 were ERT-experienced. The 2 groups had average ages of 45 years and 50 years, respectively. The ERT-naive group had a mean baseline estimated glomerular filtration rate (eGFR) of 91.4 mL/min/mL/1.73 m2, while the ERT-experienced group had a mean baseline eGFR of 89.2 mL/min/1.73 m2.
All of the patients were prescribed migalastat at a dose of 123 mg every other day. The patients all took the drug for at least 2 years, and up to 8.6 years. Investigators then calculated an annualized rate of change for the patients, using the Chronic Kidney Disease Epidemiology Collaboration equation.
In the ERT-naive cohort, the mean annualized rates of change from baseline were -1.6 mL/min/1.73 m2 overall. Stratified by gender, male patients had a mean annualized change rate of -1.8 mL/min/1.73 m2 and females had a change rate of -1.4 mL/min/1.73 m2.
In the RT-experienced cohort, the overall mean annualized rate of change was -1.6 mL/min/1.73 m2, -2.6 mL/min/1.73 m2for male patients, and -0.8 mL/min/1.73 m2for female patients.
This translated to a minimal annualized change in renal function from baseline.
Bichet and colleagues said the data show migalastat led to preserved renal function across a broad range of patients.
In conclusion, patients with Fabry disease and amenable GLA variants receiving long-term migalastat treatment (8.6 years) maintained renal function irrespective of treatment status, sex, or phenotype, they wrote.
Reference
Bichet DG, Torra R, Wallace E, et al. Long-term follow-up of renal function in patients treated with migalastat for Fabry disease. Mol Genet Metab Rep. Published online August 4, 2021. doi:10.1016/j.ymgmr.2021.100786
- 8: Techniques of Molecular Genetics - Biology LibreTexts - January 23rd, 2024
- Molecular Genetics and Metabolism Reports - ScienceDirect - December 18th, 2022
- Molecular Genetics and Metabolism | Journal - ScienceDirect - December 18th, 2022
- Molecular clock - Wikipedia - December 2nd, 2022
- Molecular Structure of Nucleic Acids: A Structure for Deoxyribose ... - December 2nd, 2022
- Molecular Genetics School of Graduate Studies - University of Toronto - October 13th, 2022
- Molecular pathways of major depressive disorder converge on the synapse | Molecular Psychiatry - Nature.com - October 13th, 2022
- Can artificial intelligence help identify best treatments for cancers? LSU researchers say yes - The Advocate - October 13th, 2022
- Results for the fiscal year on June 30, 2022 - Yahoo Finance - October 13th, 2022
- From the journals: MCP - ASBMB Today - October 13th, 2022
- From cave-dweller scientist to Nobel laureate - The Tribune India - October 13th, 2022
- Animal Genetics Market - Know the Revenue and Profit-Sources of the Industry - openPR - October 13th, 2022
- Molecular and Cell Biology and Genetics - Master of Science / PhD ... - September 25th, 2022
- What does the future hold for COVID-19? - UCLA Health Connect - September 25th, 2022
- Farooq Kperogi: Lies and truth about Obi, Atiku, and Tinubu - Peoples Gazette - September 25th, 2022
- Atavistik Bio Announces Formation of Scientific Advisory Board - Business Wire - September 25th, 2022
- Scientific Advances Point to Improved Understanding of Radiation Exposure - Livermore Independent - September 25th, 2022
- 19 million to investigate bold ideas in bioscience research - EurekAlert - September 25th, 2022
- Machel, Iwer among full list of National Awardees - Trinidad Guardian - September 25th, 2022
- Newly Discovered Protein Connected to Alzheimers Disease Risk - Neuroscience News - September 25th, 2022
- The Lab Report: Sharma Lab studies biodiversity of arachnids - The Badger Herald - September 16th, 2022
- Environmental Scientist Jesse Ausubel to Receive 2022 Nierenberg Prize for Science in the Public Interest - Scripps Institution of Oceanography - September 16th, 2022
- Malaria vax promising, to be cheap too - The Hans India - September 16th, 2022
- Dayton Therapeutics Discovers New Therapeutic Uses of Satraplatin for Treatment of Rare Lymphomas - StreetInsider.com - September 16th, 2022
- JNCASRs novel molecule prevents obesity in mice - The Hindu - September 16th, 2022
- Research Fellow in the Department of Medical and Molecular Genetics job with KINGS COLLEGE LONDON | 308234 - Times Higher Education - September 8th, 2022
- Seven Queen's researchers elected to the Royal Society of Canada - Queen's University - September 8th, 2022
- Molecular Biology with an Industrial Placement Year BSc - India Education Diary - September 8th, 2022
- Whole Exome Sequencing Market Projected to Reach CAGR of 19.0% Forecast by 2029, Global Trends, Size, Share, Growth, Future Scope and Key Player... - September 8th, 2022
- Molecular prevalence and genetic diversity of Bartonella spp. in stray cats of zmir, Turkey - Parasites & Vectors - Parasites & Vectors - August 30th, 2022
- How light and temperature work together to affect plant growth - EurekAlert - August 30th, 2022
- Ancient Mummies' Lousy View of the Past - The Scientist - August 30th, 2022
- 'Gray blanket' over Australia: Genetics may have aided European rabbit invasion of continent in 1859, says study - Down To Earth Magazine - August 30th, 2022
- Meet the UNC Pembroke professor leading research on Alzheimers disease - The Robesonian - August 30th, 2022
- Massive Genome Study Informs the Biology of Reading and Language - Neuroscience News - August 30th, 2022
- People in the News at Gencove, IsoPlexis, HTG Molecular, More - GenomeWeb - August 30th, 2022
- Professor (with Head of Department potential), Department of Medical & Molecular Genetics job with KINGS COLLEGE LONDON | 304203 - Times Higher... - August 14th, 2022
- Kathy C. Cordes, a former Baltimore Sun artist who later worked at NASA's Space Telescope Institute, dies Baltimore Sun - Baltimore Sun - August 14th, 2022
- Discovery of the interactions between plants and arbuscular mycorrhizal fungi - EurekAlert - August 14th, 2022
- A new drug could repair stroke damage to memory and movement - Freethink - August 14th, 2022
- CDC Says Recent Flu Season Mild, Maybe Owing to COVID Precautions - Medscape - August 14th, 2022
- Study Using NanoStrings GeoMx Digital Spatial Profiler Featured on the Cover of Nature Genetics - BioSpace - August 14th, 2022
- New virus detected in China; here's everything you need to know about it - WCCO - August 14th, 2022
- Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine... - August 14th, 2022
- Psychiatry, Fraud, and the Case for a Class-Action Lawsuit - Mad In America - Mad in America - August 14th, 2022
- Scientists Grow Mouse Embryos Without Sperm Or Eggs - EverythingGP - August 14th, 2022
- The Pitfalls of Evolutionary Genomics - SciTechDaily - August 14th, 2022
- How a simple home DNA test unravelled the genetic code that could help prolong my life - The National - August 14th, 2022
- Graduate Research Officer job with UNIVERSITY OF WESTERN AUSTRALIA | 304277 - Times Higher Education - August 14th, 2022
- These Are the Degrees of the Future - Gizmodo - August 14th, 2022
- University of Chicago obituaries - University of Chicago - August 14th, 2022
- Letter: Abortion and social planning - Daily Herald - August 14th, 2022
- Postdoctoral Researcher, Seaweed Molecular Biology, Physiology and Genetics, Ryan Institute, School job with NATIONAL UNIVERSITY OF IRELAND, GALWAY |... - August 5th, 2022
- Researchers crack 30-year-old mystery of odour switching in worms - University of Toronto - August 5th, 2022
- 1st synthetic mouse embryos complete with beating hearts and brains created with no sperm, eggs or womb - Livescience.com - August 5th, 2022
- Rallybio Appoints Wendy K. Chung, M.D., Ph.D., to Its Board of Directors - Business Wire - August 5th, 2022
- Luke Goldman: Using eDNA to save the Atlantic cod - UMaine News - University of Maine - University of Maine - August 5th, 2022
- Duke Announces Winners of the 2022 DST Spark Seed Grants - Duke Today - July 27th, 2022
- WVXU: UC scientists are deep-freezing molecules. Here's why they're so excited about it - University of Cincinnati - July 27th, 2022
- Better Diagnosis and Treatment: Genetic Clues to Age-Related Macular Degeneration - SciTechDaily - July 27th, 2022
- Regeneron Announces the 2022 Winners of the Regeneron Prize for Creative Innovation - PR Newswire - July 27th, 2022
- Replay Launches with $55 Million Seed to Reprogram Biology by Writing and Delivering Big DNA - GlobeNewswire - July 27th, 2022
- How is Biotechnology revolutionising food and beverage industry? - The Statesman - July 27th, 2022
- ACP offers guidance on the ethical use of genetic testing and precision medicine - EurekAlert - July 27th, 2022
- Barbour Appointed Dean of The Graduate School and Vice Provost for Graduate Education - Duke Today - July 19th, 2022
- UC scientists are deep-freezing molecules. Here's why they're so excited about it - WVXU - July 19th, 2022
- Research Fellow, Molecular Biology / Recombinant Protein Production and Purification (Biol Sci) job with NATIONAL UNIVERSITY OF SINGAPORE | 301436 -... - July 19th, 2022
- UF researchers discover new way to inhibit virus that causes COVID-19 - University of Florida - July 19th, 2022
- Streamlined Genomic Testing Platforms Are Taking on A Bigger Role in Cancer Care - OncLive - July 19th, 2022
- Bachelor of Science in Biology - National University - July 19th, 2022
- Genes may influence our successes and failures in life, according to Professor Kathryn Paige Harden - ABC News - July 19th, 2022
- Lecturer for Department of Experimental Biology job with MASARYK UNIVERSITY | 301048 - Times Higher Education - July 19th, 2022
- Lecturer in Biology (Education Focused) job with UNIVERSITY OF SYDNEY | 300815 - Times Higher Education - July 19th, 2022
- People's University Is All About Dinosaurs This Summer - Wheeling Intelligencer - July 19th, 2022
- Radiologists hope to use AI to improve readings - University of Miami: News@theU - July 19th, 2022
- Rare Disease Genetic Testing Market 2022 Emerging Trends, Comprehensive Study With Top Companies and Key Players till 2030 - Taiwan News - July 11th, 2022
- Proteases implicated in ulcerative colitis - ASBMB Today - July 11th, 2022
- Cancer has been a scourge for centuries, advances in science have made the disease far less intractable - The Indian Express - July 11th, 2022
- Scientists discover key genes behind insect migrations - EurekAlert - July 11th, 2022
- Neuroimaging Techniques and What a Brain Image Can Tell Us - Technology Networks - July 11th, 2022