Migalastat: First Global Approval

Drugs

Volumn 76, Issue 11, 2016, Pages 1147-1152

  Markham, Anthony ^a  

^a SPRINGER   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

CREATININE; MIGALASTAT; 1 DEOXYNOJIRIMYCIN;

ARTICLE; CLINICAL TRIAL (TOPIC); CREATININE BLOOD LEVEL; DRUG APPROVAL; EUROPEAN UNION; FABRY DISEASE; FATIGUE; HEADACHE; HEART EJECTION FRACTION; HUMAN; LONG TERM CARE; PARESTHESIA; PATENT; PHASE 2 CLINICAL TRIAL (TOPIC); SIDE EFFECT; ANALOGS AND DERIVATIVES;

1-DEOXYNOJIRIMYCIN; DRUG APPROVAL; EUROPEAN UNION; FABRY DISEASE; HUMANS; PATENTS AS TOPIC;

EID: 84976448052     PISSN: 00126667     EISSN: 11791950     Source Type: Journal    
DOI: 10.1007/s40265-016-0607-y     Document Type: Article

References (14)

1. Amicus Therapeutics. Amicus Therapeutics announces European Commission approval for Galafold™ (migalastat) in patients with Fabry disease in European Union [media release]. 31 May 2016. http://www.amicustherapeutics.com.
2. European Medicines Agency. Galafold 123 mg hard capsules: summary of product characteristics. 2016. http://www.ema.europa.eu/. Accessed 22 June 2016.
3. Benjamin ER, Khanna R, Schilling A, et al. Co-administration with the pharmacological chaperone AT1001 increases recombinant human alpha-galactosidase A tissue uptake and improves substrate reduction in Fabry mice. Mol Ther. 2012;20(4):717–26.
4. Germain DP, Giugliani R, Hughes DA, et al. Safety and pharmacodynamic effects of a pharmacological chaperone on alpha-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis. 2012;7:91.
5. Johnson FK, Mudd PN, Janmohamed SG. Relative bioavailability and the effect of meal type and timing on the pharmacokinetics of migalastat in healthy volunteers. Clin Pharmacol Drug Dev. 2015;4(3):193–202.
6. Mudd PN, Johnson FK, Churchill A. A phase 1 study to investigate the absorption, metabolism and excretion of [14C] migalastat hydrochloride following a single oral administration in healthy volunteers [abstract no. 1699358]. Clin Pharmacol Drug Dev. 2013;2(Suppl 1):18–9.
7. Johnson FK, Mudd PN Jr, Sitaraman S, et al. An open-label study to determine the safety and pharmacokinetics of AT1001 in subjects with impaired renal function and healthy subjects with normal renal function [abstract no. PII-92]. Clin Pharmacol Ther. 2013;93(Suppl 1):S85.
8. Benjamin ER, Hamler R, Brignol N, et al. Migalastat reduces plasma globotriaosylsphingosine (lyso-Gb3) in Fabry patients: results from the FACETS phase 3 study [abstract no. P-448]. J Inherit Metab Dis. 2014;37(Suppl 1):S161.
9. Germain DP, Bichet DG, Giugliani R, et al. Subjects treated with migalastat continue to demonstrate stable renal function and reduced left ventricular mass index over 3 years in a long-term extension study of Fabry disease [abstract no. O-050]. J Inherit Metab Dis. 2015;38(Suppl 1):S56.
10. Germain DP, Bichet D, Giugliani R, et al. Treatment with migalastat results in reduced levels of disease substrate and stable renal function in a phase 3 study of Fabry disease [abstract no. O-060]. J Inherit Metab Dis. 2014;37(Suppl 1):S43.
11. Schiffmann R, Bichet DG, Germain DP, et al. Improvement in gastrointestinal symptoms observed in the phase 3 FACETS (AT1001-011) study of migalastat in patients affected with Fabry disease [abstract plus poster]. Mol Genet Metab. 2015;114(2):S103–4.
12. Hughes D, Bichet DG, Giugliani R, et al. Long-term efficacy and safety of migalastat compared to enzyme replacement therapy in Fabry disease: phase 3 study results [abstract no. 115]. Mol Genet Metab. 2015;114(2):S57.
13. Giugliani R, Waldek S, Germain DP, et al. A phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects. Mol Genet Metab. 2013;109(1):86–92.
14. Warnock DG, Bichet DG, Holida M, et al. Oral migalastat HCl leads to greater systemic exposure and tissue levels of active alpha-galactosidase A in fabry patients when co-administered with infused agalsidase. PLoS One. 2015;10(8):e0134341.