title: Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
abstract: Gaucher disease is the first lysosomal disorder for which clinically effective enzyme replacement therapy has been introduced. Lifelong treatment with imiglucerase, the recombinant glucocerebrosidase manufactured by the Genzyme Corporation (MA, USA), is administered intravenously - usually at biweekly intervals. An acute shortage of imiglucerase (to 20% of prior global supply) has occurred as a result of viral contamination of the production facility; production was halted, and a full supply of imiglucerase is not anticipated until January 2010. An urgent meeting of physicians, researchers, and patients was convened through the agency of the European Working Group for Gaucher Disease; this was instigated by patients internationally represented by the European Gaucher Alliance. Here we present a position statement based on the findings of the group, with key recommendations about identification and monitoring of at-risk patients threatened by the abrupt withdrawal of treatment, the equitable distribution of residual imiglucerase - and access to alternative treatments including those that have completed phase III clinical trials but have not yet been licensed.
authors.affiliation: Department of Endocrinology and Metabolism, Academic Medical Centre, F5-170, PO Box 22660, 1100 DD Amsterdam, The Netherlands. c.e.hollak@amc.uva.nl
authors.forename: Carla E M
authors.initials: CE
authors.lastname: Hollak
authors.forename: Stephan
authors.initials: S
authors.lastname: vom Dahl
authors.forename: Johannes M F G
authors.initials: JM
authors.lastname: Aerts
authors.forename: Nadia
authors.initials: N
authors.lastname: Belmatoug
authors.forename: Bruno
authors.initials: B
authors.lastname: Bembi
authors.forename: Yossi
authors.initials: Y
authors.lastname: Cohen
authors.forename: Tanya
authors.initials: T
authors.lastname: Collin-Histed
authors.forename: Patrick
authors.initials: P
authors.lastname: Deegan
authors.forename: Laura
authors.initials: L
authors.lastname: van Dussen
authors.forename: Pilar
authors.initials: P
authors.lastname: Giraldo
authors.forename: Eugen
authors.initials: E
authors.lastname: Mengel
authors.forename: Helen
authors.initials: H
authors.lastname: Michelakakis
authors.forename: Jeremy
authors.initials: J
authors.lastname: Manuel
authors.forename: Martin
authors.initials: M
authors.lastname: Hrebicek
authors.forename: Rosella
authors.initials: R
authors.lastname: Parini
authors.forename: Jörg
authors.initials: J
authors.lastname: Reinke
authors.forename: Maja
authors.initials: M
authors.lastname: di Rocco
authors.forename: Miguel
authors.initials: M
authors.lastname: Pocovi
authors.forename: Maria Clara
authors.initials: MC
authors.lastname: Sa Miranda
authors.forename: Anna
authors.initials: A
authors.lastname: Tylki-Szymanska
authors.forename: Ari
authors.initials: A
authors.lastname: Zimran
authors.forename: Timothy M
authors.initials: TM
authors.lastname: Cox
mesh_terms: D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives / Q000627:therapeutic use; D000352:Africa, Northern / Q000453:epidemiology; D057176:Compassionate Use Trials; D004340:Drug Contamination / Q000517:prevention & control; D015507:Drugs, Investigational / Q000600:supply & distribution / Q000627:therapeutic use; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D056947:Enzyme Replacement Therapy / Q000706:statistics & numerical data; D004866:Equipment Contamination; D005060:Europe / Q000453:epidemiology; D005776:Gaucher Disease / Q000188:drug therapy* / Q000453:epidemiology / Q000628:therapy; D005962:Glucosylceramidase / Q000600:supply & distribution* / Q000627:therapeutic use; D017408:Guidelines as Topic; D015276:Health Care Rationing; D006292:Health Priorities; D006801:Humans; D007391:International Cooperation; D008877:Middle East / Q000453:epidemiology; D011994:Recombinant Proteins / Q000600:supply & distribution / Q000627:therapeutic use; D029342:Vesivirus
chemical_list: D015507:Drugs, Investigational; D004791:Enzyme Inhibitors; D011994:Recombinant Proteins; D017485:1-Deoxynojirimycin; C059896:miglustat; D005962:Glucosylceramidase; C090568:imiglucerase