As Beutler commented in a paper written shortly after CerezymeA(r) received FDA approval, as ERT for Gaucher Disease "The high cost of
alglucerase has created difficult problems for patients in developed countries and impossible ones in under-developed countries".15 Children in the UK are relatively fortunate in that treatment for IMD (neurometabolic disorders, mitochondrial disorders and congenital disorders of glycosylation) is funded centrally as a 'Highly Specialized Services' (AGNSS).16
Global Markets Direct's, 'Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Pipeline Review, H1 2016', provides in depth analysis on Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted pipeline therapeutics.
The report provides comprehensive information on the Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45), targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.
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* The report reviews Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
Specific therapy for the nonneuronopathic manifestations of Gaucher disease has been available since 1991 firstly in the form of the macrophage targeted placenta-derived glucocerebrosidase (alglucerase, Ceredase [R], Genzyme Corporation, MA) (7), and subsequently (1994 in USA and 1997 in Europe) by recombinant human enzyme, imiglucerase (Cerezyme[R], Genzyme Corporation, MA) (8).
The treatment has been related to a reduced risk of spontaneous abortion in women treated with alglucerase and/or imiglucerase, reduced risk of Gaucher-related complications during delivery, and a reduced risk of Gaucher-related complications during the postpartum period (11).
Imiglucerase and alglucerase are generally well tolerated in Gaucher patients and have an excellent safety record (18).
Even when during breastfeeding, the enzyme was likely to be digested in the child's gastrointestinal tract suggesting minimal risk to infants (24), a continued healthy development of children breastfed by alglucerase or imiglucerase treated mothers has been reported (25).
Pulmonary hypertension developing after
alglucerase therapy in two patients with type 1 Gaucher disease complicated by the hepa-topulmonary syndrome.
Enzyme replacement with modified placental human glucocerebrosidase (
alglucerase) is an effective means of treating patients with type 1 disease.[4]
This therapy uses either imiglucerase or
alglucerase, both of which are forms of the enzyme glucocerebrosidase.