Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
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.
Hollak
Carla E M
CE
Department of Endocrinology and Metabolism, Academic Medical Centre, F5-170, PO Box 22660, 1100 DD Amsterdam, The Netherlands. c.e.hollak@amc.uva.nl
vom Dahl
Stephan
S
Aerts
Johannes M F G
JM
Belmatoug
Nadia
N
Bembi
Bruno
B
Cohen
Yossi
Y
Collin-Histed
Tanya
T
Deegan
Patrick
P
van Dussen
Laura
L
Giraldo
Pilar
P
Mengel
Eugen
E
Michelakakis
Helen
H
Manuel
Jeremy
J
Hrebicek
Martin
M
Parini
Rosella
R
Reinke
Jörg
J
di Rocco
Maja
M
Pocovi
Miguel
M
Sa Miranda
Maria Clara
MC
Tylki-Szymanska
Anna
A
Zimran
Ari
A
Cox
Timothy M
TM
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
D015507:Drugs, Investigational; D004791:Enzyme Inhibitors; D011994:Recombinant Proteins; D017485:1-Deoxynojirimycin; C059896:miglustat; D005962:Glucosylceramidase; C090568:imiglucerase