Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage

Gabor E Linthorst; Alessandro P Burlina; Franco Cecchi; Timothy M Cox; Janice M Fletcher; Ulla Feldt-Rasmussen; Roberto Giugliani; Carla E M Hollak; Gunnar Houge; Derralynn Hughes; Iikka Kantola; Robin Lachmann; Monica Lopez; Alberto Ortiz; Rossella Parini; Alberto Rivera; Arndt Rolfs; Uma Ramaswami; Einar Svarstad; Camilla Tondel; Anna Tylki-Szymanska; Bojan Vujkovac; Steven Waldek; Michael West; F Weidemann; Atul Mehta

doi:10.1007/8904_2012_160

Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage

Gabor E Linthorst, Alessandro P Burlina, Franco Cecchi, Timothy M Cox, Janice M Fletcher, Ulla Feldt-Rasmussen, Roberto Giugliani, Carla E M Hollak, Gunnar Houge, Derralynn Hughes, Iikka Kantola, Robin Lachmann, Monica Lopez, Alberto Ortiz, Rossella Parini, Alberto Rivera, Arndt Rolfs, Uma Ramaswami, Einar Svarstad, Camilla TondelAnna Tylki-Szymanska, Bojan Vujkovac, Steven Waldek, Michael West, F Weidemann, Atul Mehta

9 Citations (Scopus)

Abstract

The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.

Original language	English
Journal	JIMD Reports
Volume	8
Pages (from-to)	51-6
Number of pages	6
DOIs	https://doi.org/10.1007/8904_2012_160
Publication status	Published - 2013

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Linthorst, G. E., Burlina, A. P., Cecchi, F., Cox, T. M., Fletcher, J. M., Feldt-Rasmussen, U., Giugliani, R., Hollak, C. E. M., Houge, G., Hughes, D., Kantola, I., Lachmann, R., Lopez, M., Ortiz, A., Parini, R., Rivera, A., Rolfs, A., Ramaswami, U., Svarstad, E., ... Mehta, A. (2013). Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage. JIMD Reports, 8, 51-6. https://doi.org/10.1007/8904_2012_160

Linthorst, GE, Burlina, AP, Cecchi, F, Cox, TM, Fletcher, JM, Feldt-Rasmussen, U, Giugliani, R, Hollak, CEM, Houge, G, Hughes, D, Kantola, I, Lachmann, R, Lopez, M, Ortiz, A, Parini, R, Rivera, A, Rolfs, A, Ramaswami, U, Svarstad, E, Tondel, C, Tylki-Szymanska, A, Vujkovac, B, Waldek, S, West, M, Weidemann, F & Mehta, A 2013, 'Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage', JIMD Reports, vol. 8, pp. 51-6. https://doi.org/10.1007/8904_2012_160

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title = "Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage",

abstract = "The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.",

author = "Linthorst, {Gabor E} and Burlina, {Alessandro P} and Franco Cecchi and Cox, {Timothy M} and Fletcher, {Janice M} and Ulla Feldt-Rasmussen and Roberto Giugliani and Hollak, {Carla E M} and Gunnar Houge and Derralynn Hughes and Iikka Kantola and Robin Lachmann and Monica Lopez and Alberto Ortiz and Rossella Parini and Alberto Rivera and Arndt Rolfs and Uma Ramaswami and Einar Svarstad and Camilla Tondel and Anna Tylki-Szymanska and Bojan Vujkovac and Steven Waldek and Michael West and F Weidemann and Atul Mehta",

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AU - Linthorst, Gabor E

AU - Burlina, Alessandro P

AU - Cecchi, Franco

AU - Cox, Timothy M

AU - Fletcher, Janice M

AU - Feldt-Rasmussen, Ulla

AU - Giugliani, Roberto

AU - Hollak, Carla E M

AU - Houge, Gunnar

AU - Hughes, Derralynn

AU - Kantola, Iikka

AU - Lachmann, Robin

AU - Lopez, Monica

AU - Ortiz, Alberto

AU - Parini, Rossella

AU - Rivera, Alberto

AU - Rolfs, Arndt

AU - Ramaswami, Uma

AU - Svarstad, Einar

AU - Tondel, Camilla

AU - Tylki-Szymanska, Anna

AU - Vujkovac, Bojan

AU - Waldek, Steven

AU - West, Michael

AU - Weidemann, F

AU - Mehta, Atul

PY - 2013

Y1 - 2013

N2 - The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.

AB - The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.

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DO - 10.1007/8904_2012_160

M3 - Journal article

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SN - 2192-8304

VL - 8

SP - 51

EP - 56

JO - JIMD Reports

JF - JIMD Reports

ER -

Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage

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