Latest research on Imiglucerase

Velaglucerase alfa is a gene-activated human recombinant glucocerebrosidase used for the treatment of Type 1 Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase. Additionally, Velaglucerase alfa has also been investigated for use in Type 3 Gaucher disease.

Latest findings

These orphan drugs are Imiglucerase, sapropterin dihydrochloride, recombinant coagulation Factor VIII, coagulation factor VIIa, human coagulation Factor VIII, human prothrombin complex concentrate, bosentan, Iloprost, ambrisentan, recombinant human Growth hormone, Busulfan, teniposide, Mitoxantrone, Imatinib, Dasatinib, nilotinib, meisoindigo, Arsenious Acid, homoharringtonine, rituximab, sorafenib, Danazol, Riluzole and poractant alfa. [source, 2016]
There are five orphan drugs defined as DDD by WHO, including Imiglucerase 300U, bosentan 250 mg, iloprost 50 ug, Danazol 600 mg, Riluzole 100 mg [20]. [source, 2016]
In 2009, two treatments granted Marketing Authorizations were available: enzyme replacement therapy (ERT) using Imiglucerase (Cerezyme® [Alglucerase® before 1996], Genzyme, a SANOFI company) and substrate reduction therapy (SRT) by miglustat (Zavesca®, ACTELION). [source, 2015]
The recommended posology for Imiglucerase in France at initiation of treatment is 60 U/kg/14 days [10], subsequently dosing can be individualized according to the patient’s achievement of stabilization of parameters, and the regression or normalization of signs and/or symptoms that initially led to treatment indication [8,10-17]. [source, 2015]
Temporary manufacturing difficulties encountered at Genzyme’s manufacturing plant led to a worldwide significantly constrained Imiglucerase supply between June 2009 and November 2010. [source, 2015]
Treatment recommendations were implemented with the European Medicines Agency (EMA); high-risk groups were identified and prioritized to receive Imiglucerase and included infants, children, adolescents and adult patients at high risk for the development of severe, life-threatening disease progression [19,20]. [source, 2015]
The management of the Imiglucerase supply constraint in France proceeded in several steps: first, a recommendation of a 50% dose reduction for patients with moderate disease (June 2009), followed by a subsequent recommendation of a 50% dose reduction in children, adolescents, GD3 patients, and pregnant women, and a switch to miglustat or a discontinuation of Imiglucerase for other patients (Aug 2009). [source, 2015]
In September 2010, a letter from the EMA (Direct Healthcare Professional Communication) informed health care professionals of an improvement in supply of Imiglucerase such that patients who were being treated with reduced doses could be returned to doses per SmPC, but patients could not be initiated on Imiglucerase or switched back to Imiglucerase. [source, 2015]
Resumption of Imiglucerase production in October 2010 allowed for treatment to be progressively resumed or doses to be adjusted to individual requirements. [source, 2015]
The goal of this study was to evaluate the clinical and biological impact of the Imiglucerase supply constraint in France from 01 June 2009 to 31 October 2010 (17 months). [source, 2015]