CRANBURY, N.J., Aug. 31, 2016 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that 7 posters highlighting its Fabry program will be included in the Society for the Study of Inborn Errors of Metabolism’s 2016 SSIEM Annual Symposium to be held September 6-9, 2016 in Rome, Italy.
Poster Sessions: Wednesday, September 7, 2016 from 6-8pm CEST
- Efficacy and safety of migalastat, an oral pharmacological chaperone for Fabry disease: results from two randomized Phase 3 studies – Prof Roberto Giugliani, Medical Genetics Service, Clinic Hospital of Porto Alegre, Porto Alegre, Brazil (P-540)
- Comparison of α-galactosidase A activity in white blood cells of patients with Fabry disease after 2 weeks of exposure to migalastat, agalsidase beta, or agalsidase alfa -- Franklin Johnson, MS, Amicus Therapeutics, Inc, Cranbury, USA (P-543)
- Phenotype of Fabry disease in patients with mutations amenable to migalastat - Derralynn Hughes, DPhil, University College, London, UK (P-549)
- Efficacy of migalastat in the cohort of male patients with classic disease presentation of Fabry disease in a Phase 3 study - Prof DP Germain, Division of Medical Genetics, University of Versailles, Paris-Saclay University, Versailles, and Assistance Publique–Hopitaux de Paris, Paris, France (P-546)
- Migalastat improves gastrointestinal symptoms in patients with Fabry disease: results from a double-blind, placebo-controlled phase 3 trial (FACETS) – Dr. Schiffmann, Institute of Metabolic Disease, Baylor Research Institute, Dallas, USA (P-541)
- The validation of pharmacogenetics in the identification of patients with Fabry disease for treatment with migalastat – Xiaoyang Wu, PhD, Amicus Therapeutics, Inc. Cranbury, USA (P-547)
- Estimating the value of treatment for Fabry disease: a discrete choice experiment - Alasdair MacCulloch, BSc PG(cert) H Econ PhD, Amicus Therapeutics UK Limited., London, UK (P-531)
Satellite Symposium
Amicus will also be sponsoring a satellite symposium on September 7, 2016 from 7-8:30pm CEST titled “Chaperone Therapy: Escorting Fabry Disease into a New Era,” bringing leading international experts together to explore the complexity of Fabry disease and examine how an in-depth understanding of the molecular mechanisms of the disease has resulted in the development of a new, targeted treatment approach for the management of adult and adolescent patients with amenable mutations.
Important Safety Information
Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation.
- GALAFOLD is not intended for concomitant use with enzyme replacement therapy.
- GALAFOLD is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of GALAFOLD in children 0–15 years of age have not yet been established.
- No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
- There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse.
- While taking GALAFOLD, effective birth control should be used. It is not known whether GALAFOLD is excreted in human milk.
- Contraindications to GALAFOLD include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on GALAFOLD or switched to GALAFOLD.
- OVERDOSE: General medical care is recommended in the case of GALAFOLD overdose.
- The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received GALAFOLD. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2015 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
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