A multicenter, openlabel, expandedaccess study followed the safety of taliglucerase alfa, a plant cellexpressed recombinant enzyme replacement therapy ert, in adults with gaucher disease. It is intended as a substitute enzyme replacement therapy ert for the first line treatment of patients with type 1 gaucher disease. Openlabel, expanded access study of taliglucerase alfa in. Taliglucerase alfa will be supplied in 200unit vials that should be refrigerated until use. This analysis evaluated the efficacy and safety of taliglucerase alfa treatment for adult patients with stable gd in the 9. Taliglucerase alfa is a plant cell expressed recombinant form of glucocerebrosidase produced in transformed carrot root cells. This enzyme is necessary for the body to use fats correctly, and fats will build up in certain areas of the body if the enzyme is not present.
Elelyso should be reconstituted with sterile water for injection and diluted with 0. Indications the indications below including fdaapproved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy. How to access a pdf or word document large file warning. Taliglucerase alfa, a proprietary plant cellexpressed recombinant form of glucosylceramidase, is being developed by protalix biotherapeutics, in collaboration. Each vial of elelyso provides 200 units of taliglucerase alfa and is intended for single use only. A16ab11 taliglucerase alfa d09675 taliglucerase alfa usaninn usp drug classification br. Taliglucerase is a manmade form of an enzyme that occurs naturally in the body. Taliglucerase alfa protalix biotherapeutics, carmiel, israel is a carrot cellexpressed human recombinant. Read plant cell expressed recombinant glucocerebrosidase taliglucerase alfa as therapy for gaucher disease in patients previously treated with imiglucerase, molecular genetics and metabolism on deepdyve, the largest online rental service for scholarly research with thousands of academic publications available at your fingertips. Elelyso should be prepared using lowproteinbinding containers and administered with a. It is derived from genetically modified carrot plant root cells. If youre interested in the scientific methods behind systematic.
Pdf taliglucerase alfa is an enzyme replacement therapy ert approved for treatment of adult and paediatric patients with. Australian public assessment report for taliglucerase alfa rpc. Clinical policies help identify whether services are medically necessary based on information. Taliglucerase alfa, the first available plant cell expressed recombinant. Elelyso taliglucerase alfa for injection physician order form phone 1855elelyso 18553535976 n fax 186675875 rd 501n please note. Taliglucerase alfa is usually given every other week.
Australian public assessment report for taliglucerase alfa rpc proprietary product name. Injection, taliglucerase alfa, 10 units hcpcs code j3060 for injection, taliglucerase alfa, 10 units as maintained by cms falls under drugs, administered by injection. Elelyso taliglucerase alfa pfizer medical information us. Velaglucerase alfa trade name vpriv, manufactured by shire plc is a hydrolytic lysosomal glucocerebrosidespecific enzyme, which is a recombinant form of glucocerebrosidase indicated as a longterm enzyme replacement therapy for those suffering of gaucher disease type 1. The partnership said that uplyso, which is called elelyso taliglucerase alfa outside of latin america, was approved by the us food and drug administration in may 2012, israels ministry of health in september 2012 and the ministry of public health in uruguay in november 2012.
This repetition of headings to form internal navigation links has no substantive legal effect. Download the pdf icon dosage and administration guide. Safety and efficacy of two dose levels of taliglucerase. However, if the volume of the reconstituted product. Enzyme replacement therapy with taliglucerase alfa. The glycans present in taliglucerase alfa rpc are typical of plant. Pdf improvement in bone marrow infiltration in patients. The drug, a recombinant glucocerebrosidase used to treat gauchers disease, was the first plantmade pharmaceutical to win approval. Each vial of elelyso taliglucerase alfa for injection provides 200 units of taliglucerase alfa and is intended for single use only. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. Attempting to open large files over the internet within the browser window may cause problems. These mannoseterminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in gaucher disease. Follow your doctors dosing instructions very carefully.
Injection, taliglucerase alfa, 10 units j3060 hcpcs codes. Taliglucerase alfa definition of taliglucerase alfa by. Taliglucerase alfa is the first us food and drug administrationapproved plant cellexpressed recombinant human protein, using carrot root cell. A plantbased expression system, using carrot root cell cultures, was developed for production of taliglucerase alfa and does not require additional processing for postproduction glycosidic modifications. Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials lipids. Velaglucerase alfa is a glycoprotein of 497 amino acids. Taliglucerase alfa is an ert approved for the treatment of adult patients with type 1 gd in. On page 5767 in the 24 november 2011 issue, there are errors in the affiliations.
The active ingredient of vpriv is velaglucerase alfa, which is produced by gene activation technology in a human fibroblast cell line. Taliglucerase alfa orphanet journal of rare diseases biomed. Talimogene laherparepvec, per 1 million plaque forming units j9325. Description taliglucerase alfa rpc is a glycosylated protein with approximately 7% of its molecular mass contributed by glycans. Elelyso taliglucerase alfa rpc 200 units powder for injection taliglucerase alfa recombinant plant carrot rpc. If data for a hazard are not included in this document there is no known information at this time. Clinical policies are one set of guidelines used to assist in administering health plan benefits, either by prior authorization or payment rules. Get emergency medical help if you have signs of an allergic reaction.
The technology transfer is intended to transfer to fiocruz the capacity and skills required for the brazilian government to construct its own manufacturing facility, at its sole expense, and to produce a sustainable, high quality and cost effective supply of alfataliglicerase taliglucerase alfa. For pediatric patients, a final volume of 100120 ml should be used. A multicenter, openlabel, expandedaccess study followed the safety of taliglucerase alfa, a plant cellexpressed recombinant enzyme replacement therapy ert, in. Longterm efficacy and safety results of taliglucerase alfa. This disease is caused by the lack of a certain enzyme in the body called glucocerebrosidase. D09675 taliglucerase alfa usaninn new drug approvals in the usa br08319. Individual results of absolute spleen volume, liver volume, platelet count, and hemoglobin concentration for adult and pediatric patients after 9 months of treatment with taliglucerase alfa. Dosing elelyso taliglucerase alfa for injection safety info. Dailymed elelyso taliglucerase alfa injection, powder. Auspar elelyso taliglucerase alfa rpc pfizer australia ltd pm200303 date of finalisation 14 october 2014. Taliglucerase alfa is an intravenous enzyme replacement therapy approved for treatment of type 1 gaucher disease gd, and is the first available plant cellexpressed recombinant therapeutic protein.
It has an identical amino acid sequence to the naturally occurring enzyme. A phase 3, multicenter, openlabel, switchover trial to. The drug, a recombinant glucocerebrosidase used to treat gauchers disease, was the first plantmade pharmaceutical to win approval by the u. Taliglucerase alfa is an enzyme replacement therapy approved for treatment of gaucher disease gd. The affiliation of the eleventh author heitner is misspelled. It is used to treat the symptoms of gauchers disease. Injection, taliglucerase alfa, 10 units j3060 hcpcs. If immediate use after reconstitution or dilution is not possible, the drug solution can be stored at 48 c for up to 24 hours. Taliglucerase alfa is a plant cellexpressed betaglucocerebrosidase approved in the united states, israel, australia, canada, and other countries for enzyme. Taliglucerase alfa medicare coverage and copay details goodrx. Elelyso taliglucerase alfa clinical studies pfizer. Full text spotlight on taliglucerase alfa in the treatment of pediatric.
Tell your caregivers right away if you have a sudden headache, dizziness, warmth or tingling, chest pain, wheezing, nausea, or a red skin rash. Taliglucerase alfa injection is used for the longterm treatment of type 1 gaucher disease. Taliglucerase alfa is a recombinant active form of the lysosomal enzyme. Policy and procedures for developing the niosh list of antineoplastic and other hazardous drugs in healthcare settings. Elelyso taliglucerase alfa is a hydrolytic lysosomal glucocerebrosidespecific enzyme indicated for longterm enzyme replacement therapy ert for adults with a confirmed diagnosis of type 1 gaucher disease. The reconstitution and dilution steps must be completed using aseptic techniques. Elelyso taliglucerase alfa for injection product monograph page.
Longterm efficacy and safety results of taliglucerase alfa through 5. New research methods resources plus a pubmed filter. Taliglucerase alfa approved for gaucher disease american. The introduction of ert has significantly impacted the treatment of type 1 gaucher disease. A link to download a pdf version of the drug profile will be included in your email receipt. Pharmacokinetics of novel plant cellexpressed taliglucerase. Taliglucerase alfa was granted orphan designation by the european commission for the treatment of gaucher disease on march 23, 2010. Taliglucerase alfa is a manmade form of an enzyme that is missing in patients with gauchers disease.
This tables of contents is a navigational tool, processed from the headings within the legal text of federal register documents. Plant cell expressed recombinant glucocerebrosidase. Pivotal trial with plant cellexpressed recombinant. They include but are not limited to policies relating to evolving medical technologies and procedures. Nov 07, 2019 you may be given other medications to prevent certain side effects of taliglucerase alfa.
Taliglucerase alfa is an enzyme replacement therapy ert approved for treatment of adult and paediatric patients with type 1 gaucher disease gd in several countries. For adult patients, a final volume of to 150 ml may be used. Elelyso taliglucerase alfa elelyso fep clinical rationale rationale for inclusion in pa program background gaucher disease is an inherited lysosomal storage disorder in humans that results in the inability to produce glucocerebrosidase, an enzyme necessary for fat metabolism. Requires prior authorization by medical director or designee.
Taliglucerase alfa doses are based on weight especially in. On march 20, 2012, the north carolina physician advisory group approved the n. Taliglucerase alfa protalix biotherapeutics, carmiel, israel is a new acid. Health choice nchc clinical coverage policy electrocardiography. Federal register niosh list of antineoplastic and other. In this report, taliglucerase alfa pharmacokinetics were assessed in adult and pediatric patients with gaucher disease from.
Your doctor may occasionally change your dose to make sure you get the best results. Taliglucerase alfa is a betaglucocerebrosidase enzyme replacement therapy approved in the united states, israel, and other countries for treatment of type 1 gaucher disease in adults, and is the first approved plant cellexpressed recombinant protein. A pfizer spokesman said taliglucerase alfa will be available from centric health resources, a specialty pharmacy provider. It is strongly recommended you download this document to your own computer and open from there. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with gaucher disease. In this report, taliglucerase alfa pharmacokinetics were assessed in adult and pediatric patients with gaucher disease from separate. Study pb06005 assessed the efficacy and safety of taliglucerase alfa at 2 dose levels, 30 ukg and. Benefit determinations are subject to applicable member contract language. Taliglucerase alfa medicare coverage and copay details. Longterm enzyme replacement therapy in patients with nonneuronopathic type 1 gaucher disease 1 5 9 designated an orphan drug by fda for this use. Taliglucerase alfa by injection treats type 1 gaucher disease.
Learn more about the pfizer rxpathways prescription program, and find out if you are eligible. Pfizer rxpathways provides access to copay and savings offers for many brandname medicines. It is used as an enzyme replacement in people with type i gaucher disease. Some people receiving a taliglucerase alfa injection have had a reaction when the medicine was injected, or up to 3 hours later. On november 30, 2009, pfizer and protalix biotherapeutics, inc. The nlm drug information portal gives users a gateway to selected drug information from the national library of medicine and other key government agencies. Quick reference guide for elelyso taliglucerase alfa for. Read download file report abuse an antitrust analysis of nba and nfl draft eligibility rules 2 had lebron been born just two years later, he 8 prior to kevin garnett in 1995, only four players were drafted by nba teams straight out of high school. The safety and efficacy of elelyso were assessed in 31 patients 26 adult and 5 pediatric patients with type 1 gaucher disease who were switched from imiglucerase to elelyso.
Jun 15, 2012 taliglucerase alfa will be supplied in 200unit vials that should be refrigerated until use. Herein, we report longterm safety and efficacy results of taliglucerase alfa in treatmentnaive adult patients with gd. Benefit determinations are subject to applicable member contract languag. Iv infusion, containing t aliglucerase alfa rpc 200 units, for the following indication. At present, available enzyme therapies include imiglucerase, velaglucerase alfa, and taliglucerase alfa, all of which are generally administered intravenously every other week 6, 8, 17. Taliglucerase alfa bluecross blueshield of tennessee. Patients previously treated on a stable dose of imiglucerase are recommended to begin treatment with elelyso at that. Nov 24, 2011 taliglucerase alfa protalix biotherapeutics, carmiel, israel is a carrot cellexpressed human recombinant.
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