Amicus Therapeutics Highlights New Fabry Program Data at WORLDSymposium™ 2017
New Phase 3 Retrospective Analysis Shows Correlation between Reduction in Disease Substrate (Kidney Interstitial Capillary GL-3) and Improved Diarrhea in Fabry Patients with Amenable Mutations treated with Migalastat
Supportive Study for Japanese New Drug Application (J-NDA) Demonstrates Migalastat Exposure is Similar in Japanese and non-Japanese Individuals
Data Highlights for Migalastat for Fabry Disease at WORLDSymposium 2017
Phase 3 Retrospective Analysis - Correlation between Substrate Reduction and Reduction in Diarrhea
In a poster1 from Study 011 (FACETS) in Fabry patients who were naïve to ERT, a retrospective analysis from baseline to month 6 demonstrated that migalastat reduces disease substrate (KIC GL-3) and improves diarrhea symptoms (GSRS-D) in patients with Fabry disease with amenable
mutations. Key highlights were as follows:
- Migalastat demonstrated a statistically significant reduction in the combined endpoint of disease substrate (KIC GL-3 and diarrhea (GSRS-D) (p=0.009; 1-sided)
- Reduction in disease substrate was significantly correlated with an improvement in diarrhea
- 83% (15/18) of migalastat-treated patients demonstrated a reduction in disease substrate and/or improvement in diarrhea when either or both were elevated at baseline compared with 33% (5/15) of patients treated with placebo
To support full approval in the
Supportive Pharmacokinetics (PK) Study for Japanese Regulatory Submission
As previously announced, Amicus plans to submit a J-NDA in
- Exposure to migalastat was similar between Japanese and non-Japanese healthy volunteers
- The PK properties of migalastat were not different between Japanese and non-Japanese patients with Fabry disease
Migalastat is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as
amenable mutations. On
About Galafold™ and Amenable Mutations
Galafold™ (migalastat) is a first-in-class chaperone therapy approved in the EU as a monotherapy for Fabry disease in patients with amenable mutations. Galafold works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. A proprietary in vitro assay (Galafold Amenability Assay) was used to classify more than 800 known GLA mutations as "amenable" or "not amenable" to treatment with Galafold. The current EU label includes 313 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay, which represent between 35% and 50% of the currently diagnosed Fabry population.
Healthcare providers in the EU may access the website www.galafoldamenabilitytable.com to quickly and accurately identify which mutations are categorized as "amenable" or "not amenable" to Galafold. Amicus expects to submit updates to the label as additional GLA mutations are identified and tested in the Galafold Amenability Assay.
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 Fabry Disease
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset.
About
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2Hoffmann B et al. Clin Gastroenterol Hepatol.
2007;5(12):1447-1453.
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4Company filings and Amicus estimates
Forward Looking Statements
This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to data, future studies and ongoing regulatory strategies for migalastat. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "confidence," "encouraged," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the data reported herein will not be predictive of future results, that later study results will not support
further development of migalastat, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize migalastat. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended
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CONTACTS: Investors/Media:Source:Amicus Therapeutics Sara Pellegrino Senior Director, Investor Relations spellegrino@amicusrx.com (609) 662-5044 Media: MWW PRSid Dinsay sdinsay@mww.com (646) 381-9017
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