How to use taliglucerase alfa solution, reconstituted recon soln this medication is given slowly, usually over 1 to 2 hours, into a vein as directed by your doctor, usually every 2 weeks. Vpriv should be administered under the supervision of a healthcare professional. If you encounter technical difficulties in viewing our product information please contact. A multicenter extension study of taliglucerase alfa in adult. Taliglucerase alfa side effects, dosage, interactions. Taliglucerase alfa was well tolerated, and meaningful and sustained. Please contact pfizer canada medical information at 1 800 4636001 with any comments or inquiries. Taliglucerase alfa is usually given every other week. Elelyso taliglucerase alfa rpc 200 units powder for injection taliglucerase alfa recombinant plant carrot rpc. Dotted lines with open symbols correspond to patient on a dose of 30 ukg biweekly. Tell your caregivers right away if you have a sudden headache, dizziness, warmth or tingling, chest pain, wheezing, nausea, or a red skin rash. Elelyso should be reconstituted with sterile water for injection and diluted with 0. Your dose needs may change if you gain or lose weight.
Protalix announces nda submission update for taliglucerase. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with gaucher disease. Elelyso taliglucerase alfa for injection is a hydrolytic lyso somal glucocerebrosidespecific enzyme indicated for longterm enzyme replacement therapy ert for adults with a confirmed diagnosis of type 1 gaucher disease. Elelyso taliglucerase alfa for injection is indicated for the treatment of patients with a confirmed diagnosis of type 1 gaucher disease. Taliglucerase alfaelelyso generic is an enzyme, prescribed for type 1 gaucher disease. This is a category 1 submission to register elelyso taliglucerase alfa for the treatment of systemic symptoms in patients with gaucher disease.
Elelyso for gaucher disease protalix biotherapeutics. Taliglucerase alfa elelyso national drug monograph. 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. Taliglucerase alfa differs from native human glucocerebrosidase by two amino acids at the n terminal and up to 7 amino acids at the c terminal. A multicenter extension study of taliglucerase alfa in adult subjects with gaucher disease. 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. Vpriv velaglucerase alfa for injection is a hydrolytic lysosomal glucocerebrosidespecific enzyme indicated for longterm enzyme replacement therapy ert for pediatric and adult patients with type 1 gaucher disease 1. The most serious side effects in patients treated with vpriv were hypersensitivity reactions. For more information, please call 844 9897284 or visit eligible patients can register for valuable savings offers for nearly 40 brand name medications. Taliglucerase alfa injection is used for the longterm treatment of type 1 gaucher disease. Taliglucerase alfa has been the first biotherapeutic expressed in plant cells to be approved by.
Fda issues complete response letter for taliglucerase alfa. To report an adverse event, please call 18004381985. A multicenter extension study of taliglucerase alfa in. Vpriv is given every other week by intravenous infusion that typically takes up to 60 minutes.
Fda approved indications elelyso is indicated for the treatment of patients with a confirmed diagnosis of type 1 gaucher disease gd1. Taliglucerase alfa, a proprietary plant cellexpressed recombinant form of glucosylceramidase, is being developed by protalix biotherapeutics, in collaboration. Safety and efficacy of two dose levels of taliglucerase alfa. Taliglucerase alfa, a hydrolytic lysosomal glucocerebrosidespecific enzyme for intravenous infusion, is a recombinant active form of the lysosomal enzyme. On 3 july 2012, the committee for medicinal products for human use chmp recommended the refusal of a marketing. Taliglucerase alfa medicare coverage and copay details goodrx. Elelyso taliglucerase alfa for treating gaucher disease clinical. Elelyso taliglucerase alfa adverse reactions pfizer. Injection, taliglucerase alfa, 10 units hcpcs code j3060 for injection, taliglucerase alfa, 10 units as maintained by cms falls under drugs, administered by injection. The approval for taliglucerase alfa was based on data from the following clinical trials in patients with type 1 gaucher disease. Indications the indications below including fdaapproved indications and compendial uses are considered a.
Taliglucerase alfa indications, dosage, side effects and. Predicted amino acid sequence for taliglucerase alfa rpc. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. B indications longterm replacement therapy in adults with confirmed type 1 gaucher disease. Injection, taliglucerase alfa, 10 units j3060 hcpcs. The safety of elelyso at dosages of either 30 unitskg n16 or 60 unitskg n16 every other week was assessed in 32 adult treatmentnaive patients aged 19 to 74 years with type 1 gaucher disease in a 9month doubleblind, randomized clinical trial. D09675 taliglucerase alfa usaninn new drug approvals in the usa br08319.
It is used to treat the symptoms of gauchers disease. Please consult a qualified health professional for more detailed information. None of these is ab le to cross the blo odbrain barri er bbb, being ineffective in neurological symptoms 4. Elelyso taliglucerase alfa is an intravenous drug indicated for the treatment of type one gaucher. Policycriteria provider must submit documentation such as office chart notes, lab results or other clinical. Quick reference guide for elelyso taliglucerase alfa for. Taliglucerase alfa doses are based on weight especially in children and teenagers. Vpriv velaglucerase alfa for the treatment of gaucher disease gd for when appropriate criteria are met. Taliglucerase alfa is a plantcell expressed form of glucocerebrosidase gcd. It helps to decrease the harmful effects of the disease by breaking down glucocerebroside. For 2016, elelyso was ranked third for pharmaceuticals with. The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Feb 02, 2010 protalix announces nda submission update for taliglucerase alfa for the treatment of gaucher disease feb.
Each vial of elelyso contains 200 units of taliglucerase alfa rpc, 195 mg mannitol. Part b drugs is outlined in the medicare benefit policy manual. An enzyme replacement therapy using plant cell expression technology gaucher disease gd is a rare, genetic lysosomal. However, if the volume of the reconstituted product. Vpriv velaglucerase alfa for injection is a prescription medication indicated for longterm enzyme replacement therapy ert for patients with type 1 gaucher disease important safety information. Pdf parkinsonisms and glucocerebrosidase deficiency. Taliglucerase alfa medicare coverage and copay details. Quest integration medicare advantage original effective date.
Thus, the availability of new enzymatic preparations, velaglucerase alfa vpriv and taliglucerase alfa uplyso, as well as alternative modalities such as substrate reduction and. Longterm efficacy and safety results of taliglucerase. Taliglucerase alfa definition of taliglucerase alfa by. Safety and efficacy of two dose levels of taliglucerase. Dosing elelyso taliglucerase alfa for injection safety. In 2009 protalix entered into a collaboration agreement with pfizer inc.
Elelyso taliglucerase alfa for injection physician order form phone 1855elelyso 18553535976 n fax 186675875 rd 501n please note. Hypersensitivity reactions, including serious allergic reactions anaphylaxis have occurred. Safety and efficacy across 6 clinical studies in adults and children with gaucher disease article pdf available in orphanet journal of rare diseases 1 december 2018. Your doctor may occasionally change your dose to make sure you get the best results.
The nlm drug information portal gives users a gateway to selected drug information from the national library of medicine and other key government agencies. Nov 09, 2018 taliglucerase alfa is injected into a vein through an iv. Herein, we report longterm safety and efficacy results of taliglucerase alfa in treatmentnaive adult patients with gd. Elelyso is administered intravenously through an injection. Elelyso, inntaliglucerase alfa european medicines agency. Pfizer rxpathways connects eligible patients, regardless of their insurance status, to a range of assistance programs that offer insurance support, copay help, and medicines for free or at a savings. Adjust dosage based on achievement and maintenance of the patients therapeutic goals. 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 intravenous enzyme replacement therapy approved for treatment of type 1 gaucher disease gd, and is the first available plant cellexpressed recombinant therapeutic protein. Nov 07, 2019 taliglucerase is used as an enzyme replacement in people with type i gaucher disease in adults and children at least 4 years old. A multicenter extension study of taliglucerase alfa in adult subjects with gaucher disease the safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Taliglucerase alfa 30 ukg and 60 ukg were efficacious in treatmentnaive adults in study pb06001 and in treatmentnaive pediatric patients in study pb06005. Product monograph download pdf, 691kb patient information download pdf, 224kb canadian regulations limit the scope of information we are permitted to give on prescription drugs via the internet or other means. Carmiel, israel pr newswire protalix biotherapeutics, inc. Canadian regulations limit the scope of information we are permitted to give on prescription drugs via the internet or other means. Enzyme replacement therapy for gaucher disease cerezyme. This enzyme is necessary for the body to use fats correctly, and fats will build up in certain areas of the body if. Action prevents the accumulation of glucocerebrosides in cells. 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. These highlights do not include all the information needed to use vpriv safely and effectively. Taliglucerase alfa is the first plant cellbased recombinant therapeutic protein approved by the fda or any other major regulatory authority. For adult patients, a final volume of to 150 ml may be used.
Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials lipids. Prior approval is required to ensure the safe, clinically appropriate and cost effective use of elelyso while maintaining optimal therapeutic outcomes. Dosage and administration 60 unitskg administered every other week as a 60minute intravenous. A healthcare provider will give you this injection.
Each vial of elelyso taliglucerase alfa for injection provides 200 units of taliglucerase alfa and is intended for single use only. Patients currently receiving imiglucerase can be switched to taliglucerase alfa at the same. It has an identical amino acid sequence to the naturally occurring enzyme. Elelyso taliglucerase alfa rpc 200 units powder for injection. Taliglucerase alfa, commercially known as elelyso, is a biopharmaceutical drug developed by protalix and pfizer. The glycans present in taliglucerase alfa rpc are typical of plant.
Taliglucerase alfa is a manmade form of an enzyme that is missing in patients with gauchers disease. Jan 03, 2017 elelyso taliglucerase alfa for intravenous injection. Patients currently receiving imiglucerase can be switched to taliglucerase alfa at the same dosage. Longterm efficacy and safety results of taliglucerase alfa. Appropriate medical support should be available when vpriv is administered. Taliglucerase alfa elelyso is a hydrolytic lysosomal glucocerebrosidespecific enzyme. Elelyso taliglucerase alfa for injection is a hydrolytic lysosomal glucocerebrosidespecific enzyme indicated for longterm enzyme replacement therapy. The medicine has been developed as an enzyme replacement therapy by p rotalix limited, and pfizer australia pl is the australian sponsor. Changes in bone marrow fat fractions ff, hb level, platelet count, liver volume, spleen volume and chitotriosidase activity in taliglucerase alfa treated patients. This disease is caused by the lack of a certain enzyme in the body called glucocerebrosidase.
Taliglucerase alfa is an enzyme replacement therapy ert approved for treatment of adult and paediatric patients with type 1 gaucher. Elelyso, a long term enzyme replacement therapy, is a recombinant analog of human lysosomal. Taliglucerase alfa solution, reconstituted recon soln side effects by likelihood and severity common side effects if experienced, these tend to have a less severe expression. Listing a study does not mean it has been evaluated by the u. You may be given other medications to prevent certain side effects of taliglucerase alfa. Taliglucerase alfa leads to favorable bone marrow responses. Follow your doctors dosing instructions very carefully.
Food and drug administration fda issued a complete response letter crl regarding the companys new drug application nda for taliglucerase alfa for the treatment of gaucher disease. Description taliglucerase alfa rpc is a glycosylated protein with approximately 7% of its molecular mass contributed by glycans. Common and rare side effects for taliglucerase alfa. Elelyso taliglucerase alfa elelyso fep clinical rationale patients younger than 4 years of age 1. Not known whether taliglucerase alfa is distributed into milk. Some people receiving a taliglucerase alfa injection have had a reaction when the medicine was injected, or up to 3 hours later. Please see ful important safet information on page 6 nd ful prescribin information startin on page 7.
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