Taliglucerase alfa is a plant cell expressed recombinant form of glucocerebrosidase produced in transformed carrot root cells. Each vial of elelyso provides 200 units of taliglucerase alfa and is intended for single use only. Taliglucerase alfa is the first us food and drug administrationapproved plant cellexpressed recombinant human protein, using carrot root cell. Taliglucerase alfa is a manmade form of an enzyme that is missing in patients with gauchers disease. Taliglucerase alfa by injection treats type 1 gaucher disease. This disease is caused by the lack of a certain enzyme in the body called glucocerebrosidase. Elelyso taliglucerase alfa pfizer medical information us. A multicenter, openlabel, expandedaccess study followed the safety of taliglucerase alfa, a plant cellexpressed recombinant enzyme replacement therapy ert, in adults with gaucher disease. Pfizer inc believes that the information contained in this material safety data sheet is accurate, and while it is provided in good faith, it is without warranty of any kind, expressed or implied. If data for a hazard are not included in this document there is no known information at this time.
Taliglucerase alfa bluecross blueshield of tennessee. Openlabel, expanded access study of taliglucerase alfa in. Enzyme replacement therapy with taliglucerase alfa. Pfizer rxpathways provides access to copay and savings offers for many brandname medicines. On march 20, 2012, the north carolina physician advisory group approved the n. Elelyso taliglucerase alfa rpc 200 units powder for injection taliglucerase alfa recombinant plant carrot rpc.
Taliglucerase alfa is usually given every other week. How to access a pdf or word document large file warning. Elelyso taliglucerase alfa for injection physician order form phone 1855elelyso 18553535976 n fax 186675875 rd 501n please note. Herein, we report longterm safety and efficacy results of taliglucerase alfa in treatmentnaive adult patients with gd. Attempting to open large files over the internet within the browser window may cause problems.
The glycans present in taliglucerase alfa rpc are typical of plant. 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 analysis evaluated the efficacy and safety of taliglucerase alfa treatment for adult patients with stable gd in the 9. If immediate use after reconstitution or dilution is not possible, the drug solution can be stored at 48 c for up to 24 hours. It is derived from genetically modified carrot plant root cells. A link to download a pdf version of the drug profile will be included in your email receipt. The drug, a recombinant glucocerebrosidase used to treat gauchers disease, was the first plantmade pharmaceutical to win approval by the u. This repetition of headings to form internal navigation links has no substantive legal effect. Quick reference guide for elelyso taliglucerase alfa for. Pharmacokinetics of novel plant cellexpressed taliglucerase. Iv infusion, containing t aliglucerase alfa rpc 200 units, for the following indication. Dosing elelyso taliglucerase alfa for injection safety info. Safety and efficacy of two dose levels of taliglucerase. 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.
Velaglucerase alfa is a glycoprotein of 497 amino acids. Talimogene laherparepvec, per 1 million plaque forming units j9325. Clinical policies help identify whether services are medically necessary based on information. Get emergency medical help if you have signs of an allergic reaction. For pediatric patients, a final volume of 100120 ml should be used. The affiliation of the eleventh author heitner is misspelled. Taliglucerase alfa approved for gaucher disease american. Elelyso should be reconstituted with sterile water for injection and diluted with 0. Elelyso taliglucerase alfa for injection product monograph page.
D09675 taliglucerase alfa usaninn new drug approvals in the usa br08319. A multicenter, openlabel, expandedaccess study followed the safety of taliglucerase alfa, a plant cellexpressed recombinant enzyme replacement therapy ert, in. If youre interested in the scientific methods behind systematic. On page 5767 in the 24 november 2011 issue, there are errors in the affiliations. 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. 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. 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. 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. Injection, taliglucerase alfa, 10 units j3060 hcpcs. In this report, taliglucerase alfa pharmacokinetics were assessed in adult and pediatric patients with gaucher disease from.
It is used to treat the symptoms of gauchers disease. Elelyso should be prepared using lowproteinbinding containers and administered with a. These mannoseterminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in gaucher disease. Benefit determinations are subject to applicable member contract language.
The reconstitution and dilution steps must be completed using aseptic techniques. Pivotal trial with plant cellexpressed recombinant. Full text spotlight on taliglucerase alfa in the treatment of pediatric. The drug, a recombinant glucocerebrosidase used to treat gauchers disease, was the first plantmade pharmaceutical to win approval. Taliglucerase alfa orphanet journal of rare diseases biomed. Nov 07, 2019 you may be given other medications to prevent certain side effects of taliglucerase alfa. Taliglucerase alfa medicare coverage and copay details goodrx. Pdf taliglucerase alfa is an enzyme replacement therapy ert approved for treatment of adult and paediatric patients with. 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. Taliglucerase alfa, commercially known as elelyso, is a biopharmaceutical drug developed by protalix and pfizer.
In this report, taliglucerase alfa pharmacokinetics were assessed in adult and pediatric patients with gaucher disease from separate. Auspar elelyso taliglucerase alfa rpc pfizer australia ltd pm200303 date of finalisation 14 october 2014. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. Taliglucerase alfa protalix biotherapeutics, carmiel, israel is a carrot cellexpressed human recombinant.
The nlm drug information portal gives users a gateway to selected drug information from the national library of medicine and other key government agencies. Follow your doctors dosing instructions very carefully. Taliglucerase alfa is a recombinant active form of the lysosomal enzyme. 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. It is intended as a substitute enzyme replacement therapy ert for the first line treatment of patients with type 1 gaucher disease. Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials lipids. Taliglucerase alfa will be supplied in 200unit vials that should be refrigerated until use. Australian public assessment report for taliglucerase alfa rpc proprietary product name. Clinical policies are one set of guidelines used to assist in administering health plan benefits, either by prior authorization or payment rules. Taliglucerase alfa medicare coverage and copay details. Replacement, modifiers, treatment taliglucerase alfa d09675 taliglucerase alfa usaninn targetbased classification of drugs br. Plant cell expressed recombinant glucocerebrosidase. It is used as an enzyme replacement in people with type i gaucher disease.
Longterm safety and efficacy of taliglucerase alfa in pediatric. 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. Federal register niosh list of antineoplastic and other. 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. Elelyso taliglucerase alfa clinical studies pfizer. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with gaucher disease. Taliglucerase alfa, the first available plant cell expressed recombinant. Longterm efficacy and safety results of taliglucerase alfa through 5.
Jun 15, 2012 taliglucerase alfa will be supplied in 200unit vials that should be refrigerated until use. Longterm enzyme replacement therapy in patients with nonneuronopathic type 1 gaucher disease 1 5 9 designated an orphan drug by fda for this use. A pfizer spokesman said taliglucerase alfa will be available from centric health resources, a specialty pharmacy provider. For adult patients, a final volume of to 150 ml may be used. 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. Taliglucerase alfa protalix biotherapeutics, carmiel, israel is a new acid. However, if the volume of the reconstituted product. Injection, taliglucerase alfa, 10 units j3060 hcpcs codes. 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. Taliglucerase alfa doses are based on weight especially in. Taliglucerase is a manmade form of an enzyme that occurs naturally in the body.
Your doctor may occasionally change your dose to make sure you get the best results. 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. Benefit determinations are subject to applicable member contract languag. New research methods resources plus a pubmed filter. Australian public assessment report for taliglucerase alfa rpc. On november 30, 2009, pfizer and protalix biotherapeutics, inc. 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. The active ingredient of vpriv is velaglucerase alfa, which is produced by gene activation technology in a human fibroblast cell line. Taliglucerase alfa is an ert approved for the treatment of adult patients with type 1 gd in. Taliglucerase alfa is a plant cellexpressed betaglucocerebrosidase approved in the united states, israel, australia, canada, and other countries for enzyme.
A16ab11 taliglucerase alfa d09675 taliglucerase alfa usaninn usp drug classification br. Longterm efficacy and safety results of taliglucerase alfa. Description taliglucerase alfa rpc is a glycosylated protein with approximately 7% of its molecular mass contributed by glycans. Nov 24, 2011 taliglucerase alfa protalix biotherapeutics, carmiel, israel is a carrot cellexpressed human recombinant. Patients previously treated on a stable dose of imiglucerase are recommended to begin treatment with elelyso at that. Taliglucerase alfa, a proprietary plant cellexpressed recombinant form of glucosylceramidase, is being developed by protalix biotherapeutics, in collaboration. Policy and procedures for developing the niosh list of antineoplastic and other hazardous drugs in healthcare settings. Learn more about the pfizer rxpathways prescription program, and find out if you are eligible. Pdf improvement in bone marrow infiltration in patients. Dailymed elelyso taliglucerase alfa injection, powder.
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. The introduction of ert has significantly impacted the treatment of type 1 gaucher disease. They include but are not limited to policies relating to evolving medical technologies and procedures. Taliglucerase alfa injection is used for the longterm treatment of type 1 gaucher disease. Injection, taliglucerase alfa, 10 units hcpcs code j3060 for injection, taliglucerase alfa, 10 units as maintained by cms falls under drugs, administered by injection. Taliglucerase alfa is an enzyme replacement therapy approved for treatment of gaucher disease gd. Requires prior authorization by medical director or designee. 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. Taliglucerase alfa was granted orphan designation by the european commission for the treatment of gaucher disease on march 23, 2010. Each vial of elelyso taliglucerase alfa for injection provides 200 units of taliglucerase alfa and is intended for single use only. Study pb06005 assessed the efficacy and safety of taliglucerase alfa at 2 dose levels, 30 ukg and. A phase 3, multicenter, openlabel, switchover trial to.
This tables of contents is a navigational tool, processed from the headings within the legal text of federal register documents. Health choice nchc clinical coverage policy electrocardiography. Velaglucerase alfa has the same amino acid sequence as the. Taliglucerase alfa definition of taliglucerase alfa by. Download the pdf icon dosage and administration guide. Tell your caregivers right away if you have a sudden headache, dizziness, warmth or tingling, chest pain, wheezing, nausea, or a red skin rash. It is strongly recommended you download this document to your own computer and open from there.
260 500 999 519 910 873 547 1196 53 101 281 1213 499 1107 934 690 652 463 796 97 1038 530 340 1351 1391 484 1125 1136 794 1451 430 597 240 980 898 952 1303 1340 1420